Ignite Creation Date:
2025-12-25 @ 4:50 AM
Ignite Modification Date:
2026-01-28 @ 4:08 PM
Study NCT ID:
NCT02246218
Status:
COMPLETED
Last Update Posted:
2024-07-01
First Post:
2014-09-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D056806', 'term': 'Urea Cycle Disorders, Inborn'}, {'id': 'D022124', 'term': 'Hyperammonemia'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C570223', 'term': 'glycerol phenylbutyrate'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@horizonpharma.com', 'phone': '866-479-6742', 'title': 'Colleen Canavan, Director', 'organization': 'Horizon Therapeutics, LLC'}, 'certainAgreement': {'otherDetails': 'Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months for Cohort of 2 Months to <2 Years Participants and 10.67 [6.142] months for Cohort of 0 Months to <2 Months Participants).', 'description': 'TEAEs (defined as AEs with an onset date on or after the first dose of study medication until study discontinuation) are presented.', 'eventGroups': [{'id': 'EG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 8, 'seriousNumAtRisk': 10, 'deathsNumAffected': 1, 'seriousNumAffected': 6}, {'id': 'EG001', 'title': 'RAVICTI: Age 0 to <2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.', 'otherNumAtRisk': 16, 'deathsNumAtRisk': 16, 'otherNumAffected': 16, 'seriousNumAtRisk': 16, 'deathsNumAffected': 0, 'seriousNumAffected': 11}], 'otherEvents': [{'term': 'Venous thrombosis limb', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Catheter site rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Device occlusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Drug withdrawal syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Medical device site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Agitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Stoma site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tibia fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Amino acid level decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ammonia increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Carbon dioxide decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Amino acid level increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Anion gap increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Blood bicarbonate decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Blood urea decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Body height below normal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Platelet count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Transaminases increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dacryostenosis congenital', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Plagiocephaly', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Thrombocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Leukocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Lymphocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Microcytic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Apnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Respiratory disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pneumothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tachypnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Use of accessory respiratory muscles', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gross motor delay', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Eye discharge', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ocular hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Excessive cerumen production', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Teething', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Post-tussive vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hepatic calcification', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vesicoureteric reflux', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nail ridging', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dermatitis diaper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 6}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Red man syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Seborrhoeic dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Torticollis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Metabolic acidosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hypophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hyperammonaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Feeding disorder of infancy or early childhood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Protein deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Croup infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Angular cheilitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Candida infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastrointestinal viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rhinovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'seriousEvents': [{'term': 'Ammonia increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cyanosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Apnoeic attack', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Status asthmaticus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Atelectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pneumatosis intestinalis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hyperammonaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Feeding disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Croup infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Peritonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rhinovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Medical device site infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Meningitis bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tracheitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Day 4', 'description': 'The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication.'}, {'type': 'PRIMARY', 'title': 'Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Day 4', 'description': 'The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants \\< 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication.'}, {'type': 'SECONDARY', 'title': 'Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.005', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 8 through up to Month 6', 'description': 'HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.', 'unitOfMeasure': 'HACs per half-year of patient exposure', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': '≥ 1 TEAE', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Related TEAE', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Serious TEAE', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Serious Related TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Fatal Outcome TEAE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 TEAE Leading to Study Discontinuation', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months).', 'description': 'An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '122.43', 'spread': '118.620', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-54.50', 'spread': '92.626', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.80', 'spread': '25.646', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-16.33', 'spread': '39.209', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-13.00', 'spread': '39.590', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.25', 'spread': '13.426', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.20', 'spread': '34.463', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30.80', 'spread': '17.092', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.20', 'spread': '23.506', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '39.00', 'spread': 'NA', 'comment': '1 participant analyzed', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '48.00', 'spread': '53.740', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '750.43', 'spread': '309.000', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-184.33', 'spread': '168.657', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-174.60', 'spread': '318.249', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-374.00', 'spread': '425.903', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-252.75', 'spread': '323.852', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-370.25', 'spread': '377.222', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-113.20', 'spread': '519.710', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-446.53', 'spread': '360.457', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-450.50', 'spread': '386.699', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-149.00', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '195.00', 'spread': '554.372', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '872.86', 'spread': '381.005', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-238.83', 'spread': '203.567', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-166.80', 'spread': '332.421', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-390.33', 'spread': '462.292', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-265.75', 'spread': '339.015', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-370.00', 'spread': '379.884', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-115.40', 'spread': '546.796', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-415.73', 'spread': '365.419', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-428.30', 'spread': '404.351', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-110.00', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '243.00', 'spread': '608.112', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '54.86', 'spread': '19.497', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.67', 'spread': '19.159', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.20', 'spread': '45.861', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-25.67', 'spread': '16.743', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-20.25', 'spread': '20.353', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-20.00', 'spread': '36.341', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-16.40', 'spread': '12.137', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.73', 'spread': '13.342', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-13.33', 'spread': '15.885', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-18.00', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.50', 'spread': '10.607', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '90.86', 'spread': '29.249', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.83', 'spread': '32.762', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.80', 'spread': '65.975', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-33.00', 'spread': '25.239', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-31.25', 'spread': '24.771', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-39.50', 'spread': '61.136', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-25.40', 'spread': '13.594', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-19.13', 'spread': '39.322', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-34.37', 'spread': '23.283', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-40.00', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.50', 'spread': '31.820', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '171.43', 'spread': '42.887', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.00', 'spread': '24.133', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '40.60', 'spread': '90.057', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-27.33', 'spread': '37.018', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-31.50', 'spread': '29.760', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-56.00', 'spread': '75.939', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-21.60', 'spread': '18.202', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-11.90', 'spread': '71.753', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-48.87', 'spread': '51.644', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-46.00', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.00', 'spread': '55.154', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point.'}, {'type': 'SECONDARY', 'title': 'Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.8107', 'spread': '2.17017', 'groupId': 'OG000'}]}]}, {'title': 'Month 1 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2385', 'spread': '0.77830', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.0249', 'spread': '0.74861', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1815', 'spread': '0.86056', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4434', 'spread': '0.94854', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1484', 'spread': '0.76293', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2497', 'spread': '0.80923', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6407', 'spread': '0.98695', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4164', 'spread': '0.80674', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2997', 'spread': '0.16959', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2038', 'spread': 'NA', 'comment': '1 participant with an assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5581', 'spread': '1.23993', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': "To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.", 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.7143', 'spread': '2.14922', 'groupId': 'OG000'}]}]}, {'title': 'Month 1 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2105', 'spread': '0.74135', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.0704', 'spread': '0.70393', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1065', 'spread': '0.70165', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3365', 'spread': '0.77672', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1043', 'spread': '0.56747', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1842', 'spread': '0.62205', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4875', 'spread': '0.86137', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2944', 'spread': '0.75133', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.3661', 'spread': '0.00932', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2214', 'spread': 'NA', 'comment': '1 participant with assessment', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4310', 'spread': '1.13140', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': 'To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.', 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Plasma Phenylbutyrate/Phenylbutyric Acid (PBA) Maximum Plasma Concentration (Cmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '42.44', 'spread': '36.715', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic (PK) Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA Minimum Plasma Concentration (Cmin) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.697', 'spread': '2.254', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC[0-last]) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '280.936', 'spread': '293.553', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA Time to Cmax (Tmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '8.383', 'spread': '4.564', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma Phenylacetate/Phenylacetic Acid (PAA) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '36.52', 'spread': '31.784', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '4.197', 'spread': '6.434', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '246.126', 'spread': '238.547', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.422', 'spread': '7.351', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma Phenylacetylglutamine (PAGN) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '62.45', 'spread': '27.281', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '20.62', 'spread': '14.529', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '583.835', 'spread': '285.241', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '6.573', 'spread': '7.181', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Hour 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3273', 'spread': '1993', 'groupId': 'OG000'}]}]}, {'title': '0.5 to 1.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4140', 'spread': '4399', 'groupId': 'OG000'}]}]}, {'title': '1.5 to 2.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3145', 'spread': '5045', 'groupId': 'OG000'}]}]}, {'title': '4 to 6 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5202', 'spread': '4547', 'groupId': 'OG000'}]}]}, {'title': '7.5 to 8.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3950', 'spread': '3068', 'groupId': 'OG000'}]}]}, {'title': '12 to 24 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7561', 'spread': '6956', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Day 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8859', 'spread': '10500', 'groupId': 'OG000'}]}]}, {'title': 'Month 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6274', 'spread': '4802', 'groupId': 'OG000'}]}]}, {'title': 'Month 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7386', 'spread': '6419', 'groupId': 'OG000'}]}]}, {'title': 'Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11456', 'spread': '14471', 'groupId': 'OG000'}]}]}, {'title': 'Month 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '21416', 'spread': '33695', 'groupId': 'OG000'}]}]}, {'title': 'Month 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6129', 'spread': '8024', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5347', 'spread': '3153', 'groupId': 'OG000'}]}]}, {'title': 'Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9357', 'spread': '7286', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2580', 'spread': '286', 'groupId': 'OG000'}]}]}, {'title': 'Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6400', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5250', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'End of trial', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25333', 'spread': '21324', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, End of Trial (up to Month 18)', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Hour 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.78', 'spread': '27.33', 'groupId': 'OG000'}]}]}, {'title': '0.5 to 1.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.50', 'spread': '3.39', 'groupId': 'OG000'}]}]}, {'title': '1.5 to 2.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.29', 'spread': '4.10', 'groupId': 'OG000'}]}]}, {'title': '4 to 6 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.60', 'spread': '2.18', 'groupId': 'OG000'}]}]}, {'title': '7.5 to 8.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.48', 'spread': '4.38', 'groupId': 'OG000'}]}]}, {'title': '12 to 24 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.31', 'spread': '2.33', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Day 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.82', 'spread': '2.21', 'groupId': 'OG000'}]}]}, {'title': 'Month 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.44', 'spread': '4.61', 'groupId': 'OG000'}]}]}, {'title': 'Month 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.69', 'spread': '1.82', 'groupId': 'OG000'}]}]}, {'title': 'Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.65', 'spread': '0.49', 'groupId': 'OG000'}]}]}, {'title': 'Month 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.14', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'Month 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.27', 'spread': '1.96', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.59', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.10', 'spread': '1.65', 'groupId': 'OG000'}]}]}, {'title': 'Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.04', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.64', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'End of trial', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.0', 'spread': '5.16', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, Month 18, End of Trial (up to Month 18)', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Rate of HACs: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.003', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 8 through up to Month 6', 'description': 'HAC is defined as having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension was calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.', 'unitOfMeasure': 'HACs per half-year of patient exposure', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': '≥ 1 TEAE', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Related TEAE', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Serious TEAE', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 Serious Related TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Fatal Outcome TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': '≥ 1 TEAE Leading to Study Discontinuation', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 10.67 [6.142] months).', 'description': 'An AE is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '84.97', 'spread': '52.086', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.81', 'spread': '76.417', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25.16', 'spread': '64.474', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '50.05', 'spread': '73.988', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.77', 'spread': '67.561', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '57.43', 'spread': '98.550', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '43.65', 'spread': '140.985', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '33.41', 'spread': '150.273', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.75', 'spread': '97.329', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25.75', 'spread': '62.660', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.50', 'spread': '21.920', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16.10', 'spread': '95.461', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '508.83', 'spread': '337.175', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '21.04', 'spread': '260.500', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-27.62', 'spread': '379.796', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-15.09', 'spread': '352.471', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-113.98', 'spread': '230.855', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-99.82', 'spread': '305.674', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-138.16', 'spread': '349.269', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-56.08', 'spread': '269.288', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-181.50', 'spread': '118.604', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-103.75', 'spread': '328.583', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-184.00', 'spread': '80.610', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-219.93', 'spread': '279.815', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. 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The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '133.67', 'spread': '253.829', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-81.91', 'spread': '273.127', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-60.80', 'spread': '278.083', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-51.66', 'spread': '269.558', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-82.82', 'spread': '224.269', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-118.55', 'spread': '311.125', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-11.85', 'spread': '40.693', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-115.09', 'spread': '338.186', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-249.50', 'spread': '483.776', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-195.75', 'spread': '496.313', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.00', 'spread': '62.225', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-82.54', 'spread': '228.107', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '181.49', 'spread': '257.948', 'groupId': 'OG000'}]}]}, {'title': 'Day 7 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-63.96', 'spread': '290.312', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-39.04', 'spread': '297.634', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-23.86', 'spread': '290.128', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-74.41', 'spread': '233.538', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-98.67', 'spread': '326.300', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.64', 'spread': '77.843', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-90.40', 'spread': '355.316', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-238.25', 'spread': '506.435', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-137.00', 'spread': '519.042', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '38.00', 'spread': '67.882', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-72.78', 'spread': '258.710', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.0544', 'spread': '1.26821', 'groupId': 'OG000'}]}]}, {'title': 'Month 1 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2158', 'spread': '1.35960', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2598', 'spread': '1.19544', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.1617', 'spread': '1.02572', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.0264', 'spread': '1.68215', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0828', 'spread': '1.14206', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0136', 'spread': '1.72106', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4614', 'spread': '1.25343', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6646', 'spread': '0.95334', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6830', 'spread': '0.55703', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3308', 'spread': '0.27572', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.7743', 'spread': '0.64962', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': "To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.", 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.1980', 'spread': '2.11774', 'groupId': 'OG000'}]}]}, {'title': 'Month 1 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2336', 'spread': '1.95603', 'groupId': 'OG000'}]}]}, {'title': 'Month 2 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2006', 'spread': '1.72135', 'groupId': 'OG000'}]}]}, {'title': 'Month 3 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2684', 'spread': '1.37517', 'groupId': 'OG000'}]}]}, {'title': 'Month 4 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2372', 'spread': '2.30831', 'groupId': 'OG000'}]}]}, {'title': 'Month 5 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1810', 'spread': '1.70024', 'groupId': 'OG000'}]}]}, {'title': 'Month 6 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2902', 'spread': '2.05956', 'groupId': 'OG000'}]}]}, {'title': 'Month 9 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1679', 'spread': '1.32407', 'groupId': 'OG000'}]}]}, {'title': 'Month 12 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1308', 'spread': '0.50371', 'groupId': 'OG000'}]}]}, {'title': 'Month 15 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1595', 'spread': '0.75833', 'groupId': 'OG000'}]}]}, {'title': 'Month 18 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1050', 'spread': '0.73521', 'groupId': 'OG000'}]}]}, {'title': 'Month 24 change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.7341', 'spread': '1.35582', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': 'To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.', 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Population: all enrolled participants who received any amount of study medication and had an assessment.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '46.2', 'spread': '49.8', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '4.8', 'spread': '4.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PBA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. 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The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.39', 'spread': '7.41', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '115.3', 'spread': '102.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '98.98', 'spread': '122.07', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '1321.18', 'spread': '1220.52', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.85', 'spread': '9.26', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '102.1', 'spread': '48.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '69.39', 'spread': '54.03', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '1384.12', 'spread': '1141.03', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg*hr/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '11.72', 'spread': '8.24', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Hour 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3530.43', 'spread': '3600.4', 'groupId': 'OG000'}]}]}, {'title': '0.5 to 1.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1828', 'spread': '2862', 'groupId': 'OG000'}]}]}, {'title': '1.5 to 2.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1746', 'spread': '1464', 'groupId': 'OG000'}]}]}, {'title': '4 to 6 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2260', 'spread': '1472', 'groupId': 'OG000'}]}]}, {'title': '7.5 to 8.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3530.43', 'spread': '3600.4', 'groupId': 'OG000'}]}]}, {'title': '12 to 24 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4404', 'spread': '3766', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Day 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4643', 'spread': '2506', 'groupId': 'OG000'}]}]}, {'title': 'Month 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4517', 'spread': '2485', 'groupId': 'OG000'}]}]}, {'title': 'Month 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4116', 'spread': '3137', 'groupId': 'OG000'}]}]}, {'title': 'Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7037', 'spread': '4493', 'groupId': 'OG000'}]}]}, {'title': 'Month 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2826', 'spread': '1543', 'groupId': 'OG000'}]}]}, {'title': 'Month 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6973', 'spread': '3682', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5883', 'spread': '3128', 'groupId': 'OG000'}]}]}, {'title': 'Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7006', 'spread': '4289', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5847', 'spread': '2992', 'groupId': 'OG000'}]}]}, {'title': 'Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3915', 'spread': '2584', 'groupId': 'OG000'}]}]}, {'title': 'End of trial', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6939', 'spread': '6581', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, End of Trial (up to Month 15)', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Hour 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.1', 'spread': '10.4', 'groupId': 'OG000'}]}]}, {'title': '0.5 to 1.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '46.2', 'spread': '58.6', 'groupId': 'OG000'}]}]}, {'title': '1.5 to 2.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '62.5', 'spread': '42.8', 'groupId': 'OG000'}]}]}, {'title': '4 to 6 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '34.6', 'spread': '56.2', 'groupId': 'OG000'}]}]}, {'title': '7.5 to 8.5 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.8', 'spread': '25.3', 'groupId': 'OG000'}]}]}, {'title': '12 to 24 hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '35.2', 'spread': '51.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}, {'type': 'SECONDARY', 'title': 'Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'classes': [{'title': 'Day 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.7', 'spread': '31.2', 'groupId': 'OG000'}]}]}, {'title': 'Month 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14.6', 'spread': '17.2', 'groupId': 'OG000'}]}]}, {'title': 'Month 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.3', 'spread': '13.3', 'groupId': 'OG000'}]}]}, {'title': 'Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14.4', 'spread': '10.7', 'groupId': 'OG000'}]}]}, {'title': 'Month 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.4', 'spread': '7.1', 'groupId': 'OG000'}]}]}, {'title': 'Month 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.2', 'spread': '14.0', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.5', 'spread': 'NA', 'comment': '1 participant assessed', 'groupId': 'OG000'}]}]}, {'title': 'Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.8', 'spread': '8.5', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.0', 'spread': '3.8', 'groupId': 'OG000'}]}]}, {'title': 'Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.9', 'spread': '0.3', 'groupId': 'OG000'}]}]}, {'title': 'End of trial', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.6', 'spread': '9.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, End of Trial (up to Month 15)', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Evaluable Population: all participants from the safety population with individual concentration-time profiles that allow computation of meaningful PK parameter values.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}, {'id': 'FG001', 'title': 'RAVICTI: Age 0 to < 2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '16'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Parent/Guardian', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Stopping Rule: Liver Transplant', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'A total of 27 participants were enrolled; 1 participant was not dosed and is not included in any analysis.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '26', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'RAVICTI: Age 2 Months to < 2 Years', 'description': 'Participants age 2 months to \\< 2 years received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}, {'id': 'BG001', 'title': 'RAVICTI: Age 0 to <2 Months', 'description': 'Participants age 0 to \\< 2 months received RAVICTI Oral Liquid administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '9.87', 'spread': '5.529', 'groupId': 'BG000'}, {'value': '0.83', 'spread': '0.697', 'groupId': 'BG001'}, {'value': '4.31', 'spread': '5.606', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-12-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-06', 'dispFirstSubmitDate': '2017-10-16', 'completionDateStruct': {'date': '2017-07-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-06-18', 'studyFirstSubmitDate': '2014-09-17', 'dispFirstSubmitQcDate': '2017-10-16', 'resultsFirstSubmitDate': '2019-01-14', 'studyFirstSubmitQcDate': '2014-09-18', 'dispFirstPostDateStruct': {'date': '2017-10-18', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-07-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-02-08', 'studyFirstPostDateStruct': {'date': '2014-09-22', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-03-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-10-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Up to Day 4', 'description': 'The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.'}, {'measure': 'Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Up to Day 4', 'description': 'The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants \\< 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment.'}], 'secondaryOutcomes': [{'measure': 'Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Day 8 through up to Month 6', 'description': 'HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.'}, {'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months).', 'description': 'An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24'}, {'measure': 'Assessment of Growth and Development: Baseline and Change From Baseline in Body Mass Index (BMI) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': "To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender."}, {'measure': 'Assessment of Growth and Development: Baseline and Change From Baseline in Body Surface Area (BSA) Z-Score Up to Month 24: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': 'To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.'}, {'measure': 'Plasma Phenylbutyrate/Phenylbutyric Acid (PBA) Maximum Plasma Concentration (Cmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA Minimum Plasma Concentration (Cmin) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC[0-last]) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA Time to Cmax (Tmax) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma Phenylacetate/Phenylacetic Acid (PAA) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma Phenylacetylglutamine (PAGN) Cmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, End of Trial (up to Month 18)'}, {'measure': 'Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 2 Months to <2 Years Participants', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, Month 18, End of Trial (up to Month 18)'}, {'measure': 'Rate of HACs: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Day 8 through up to Month 6', 'description': 'HAC is defined as having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension was calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group.'}, {'measure': 'Number of Participants With TEAEs, Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 10.67 [6.142] months).', 'description': 'An AE is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related.'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24'}, {'measure': 'Assessment of Growth and Development: Baseline and Change From Baseline in BMI Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': "To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BMI. The Z-scores are based on the World Health Organization's Child Growth Standards charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender."}, {'measure': 'Assessment of Growth and Development: Baseline and Change From Baseline in BSA Z-Score Up to Month 24: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 18, Month 24', 'description': 'To assess any effect of study drug treatment on growth, Z-scores were calculated to express the deviation from a reference population for BSA. The Z-scores are based on weight-for-length charts. Negative Z-scores indicate lower than typical for age and gender while positive scores indicate higher than typical for age and gender.'}, {'measure': 'Plasma PBA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PBA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAA Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN Cmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN Cmin on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN AUC(0-last) on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Plasma PAGN Tmax on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 4 and 6 hours, 8 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAGN Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 0.5 and 1 hour, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAGN Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, End of Trial (up to Month 15)'}, {'measure': 'Assessment of Urinary PAA Concentrations on the First Full Day of RAVICTI Dosing: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Hour 0 and between 0.5 and 1.5 hours, 1.5 and 2.5 hours, 4 and 6 hours, 7.5 and 8.5 hours, and between 12 and 24 hours after the first dose of the day on Day 1 for stable participants and on Day 2 for participants in HAC'}, {'measure': 'Assessment of Urinary PAA Concentrations Up to End of Trial: Cohort of 0 Months to <2 Months Participants', 'timeFrame': 'Day 7, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 15, End of Trial (up to Month 15)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Urea Cycle Disorder', 'UCD', 'Buphenyl', 'Glycerol Phenylbutyrate', 'Ravicti', 'Hyperammonemia', 'Sodium Phenylbutyrate', 'GPB', 'HPN-100'], 'conditions': ['Urea Cycle Disorder']}, 'referencesModule': {'references': [{'pmid': '28916119', 'type': 'RESULT', 'citation': 'Berry SA, Longo N, Diaz GA, McCandless SE, Smith WE, Harding CO, Zori R, Ficicioglu C, Lichter-Konecki U, Robinson B, Vockley J. Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years. Mol Genet Metab. 2017 Nov;122(3):46-53. doi: 10.1016/j.ymgme.2017.09.002. Epub 2017 Sep 8.'}, {'pmid': '30217721', 'type': 'RESULT', 'citation': 'Berry SA, Vockley J, Vinks AA, Dong M, Diaz GA, McCandless SE, Smith WE, Harding CO, Zori R, Ficicioglu C, Lichter-Konecki U, Perdok R, Robinson B, Holt RJ, Longo N. Pharmacokinetics of glycerol phenylbutyrate in pediatric patients 2 months to 2 years of age with urea cycle disorders. Mol Genet Metab. 2018 Nov;125(3):251-257. doi: 10.1016/j.ymgme.2018.09.001. Epub 2018 Sep 4.'}], 'seeAlsoLinks': [{'url': 'https://www.ravicti.com', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children.\n\nSubjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \\< 2 years of age with either a diagnosed or clinically suspected UCD.', 'detailedDescription': 'Study acquired from Horizon in 2024.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '2 Years', 'minimumAge': '1 Day', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male and female subjects up to 2 years of age\n* Signed informed consent by subject's parent/legal guardian\n* UCD diagnosis or suspected diagnosis of any subtype, except N-acetyl glutamate synthetase deficiency. If UCD has not been previously confirmed by genetic testing, consent must be obtained from parent/legal guardian prior to perform genetic testing. If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.\n\nExclusion Criteria:\n\n* Use of any investigational drug within 30 days of Day 1\n* Uncontrolled infection (viral or bacterial) or any other condition known to precipitate hyperammonemic crises. Once these precipitating factors are medically controlled, patients presenting in crisis are eligible.\n* Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5-20 times the upper limit of normal in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable subject\n* Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the subject at increased risk by participating in this study\n* Known hypersensitivity to phenylacetate (PAA) or phenylbutyrate (PBA)\n* Liver transplantation, including hepatocellular transplant\n* Subjects on hemodialysis at time of initiating RAVICTI\n* Subjects on RAVICTI for UCD management\n* Currently treated with Carbaglu® (carglumic acid)"}, 'identificationModule': {'nctId': 'NCT02246218', 'briefTitle': 'A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amgen'}, 'officialTitle': 'An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)', 'orgStudyIdInfo': {'id': 'HPN-100-009'}, 'secondaryIdInfos': [{'id': '2016-003460-38', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RAVICTI', 'description': 'RAVICTI Oral Liquid should be administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.', 'interventionNames': ['Drug: RAVICTI']}], 'interventions': [{'name': 'RAVICTI', 'type': 'DRUG', 'otherNames': ['HPN-100', 'Glycerol Phenylbutyrate', 'GPB'], 'armGroupLabels': ['RAVICTI']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Kaiser Permanente Regional Metabolic Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94034', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford Center for Clinical & Translational Research & Education', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': "Children's National Medical Center", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'Shands at University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '04102', 'city': 'Portland', 'state': 'Maine', 'country': 'United States', 'facility': 'Maine Medical Center', 'geoPoint': {'lat': 43.65737, 'lon': -70.2589}}, {'zip': '48201', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': "Children's Hospital of Michigan", 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'University Hospitals Case Medical Center', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': 'M5G 1Hs', 'city': 'Toronto', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'overallOfficials': [{'name': 'MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amgen'}]}, 'ipdSharingStatementModule': {'url': 'https://www.amgen.com/datasharing', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'timeFrame': 'Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.', 'ipdSharing': 'YES', 'description': 'De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.', 'accessCriteria': 'Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amgen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}