Viewing Study NCT00924703

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Ignite Creation Date: 2025-12-24 @ 7:41 PM
Ignite Modification Date: 2025-12-29 @ 3:45 PM
Study NCT ID: NCT00924703
Status: COMPLETED
Last Update Posted: 2021-10-12
First Post: 2009-06-18
Is NOT Gene Therapy: True
Has Adverse Events: True
Brief Title: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010661', 'term': 'Phenylketonurias'}], '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629004', 'term': 'pegvaliase'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Dlounsbury@bmrn.com', 'phone': '415-506-6348', 'title': 'Debra Lounsbury/Sr Principle Scientist, Clinical Sciences', 'organization': 'BioMarin Pharmaceutical Inc.'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Up to 109 months.', 'description': 'Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 4.03), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.', 'eventGroups': [{'id': 'EG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection', 'otherNumAtRisk': 68, 'deathsNumAtRisk': 68, 'otherNumAffected': 68, 'seriousNumAtRisk': 68, 'deathsNumAffected': 0, 'seriousNumAffected': 12}], 'otherEvents': [{'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 9}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 114, 'numAffected': 36}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 160, 'numAffected': 36}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 245, 'numAffected': 34}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 38, 'numAffected': 25}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 59, 'numAffected': 25}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 24, 'numAffected': 11}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 83, 'numAffected': 22}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 76, 'numAffected': 18}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 25, 'numAffected': 16}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 66, 'numAffected': 18}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 24, 'numAffected': 17}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Peripheral swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 18, 'numAffected': 9}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 22, 'numAffected': 15}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 20, 'numAffected': 13}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 10, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site nodule', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 17, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 253, 'numAffected': 43}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 61, 'numAffected': 26}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 59, 'numAffected': 24}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 17, 'numAffected': 12}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 41, 'numAffected': 14}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 10, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 30, 'numAffected': 14}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 16, 'numAffected': 13}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 11, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 6}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Disturbance in attention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Rash generalised', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 17, 'numAffected': 6}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 14, 'numAffected': 11}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 10, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Tendonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dermatitis contact', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 20, 'numAffected': 11}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dry skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 8}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Thermal burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 11, 'numAffected': 9}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 124, 'numAffected': 38}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 294, 'numAffected': 33}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 69, 'numAffected': 27}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 31, 'numAffected': 14}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Rash erythematous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 21, 'numAffected': 16}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 14, 'numAffected': 12}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Rash pruritic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 137, 'numAffected': 45}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 157, 'numAffected': 34}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 43, 'numAffected': 22}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 21, 'numAffected': 14}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 21, 'numAffected': 12}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 18, 'numAffected': 13}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 14, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 16, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Fungal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Microalbuminuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 4}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Amino acid level decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 52, 'numAffected': 33}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 82, 'numAffected': 33}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 41, 'numAffected': 25}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Sinus congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 33, 'numAffected': 15}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 18, 'numAffected': 10}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pulmonary congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': "Raynaud's phenomenon", 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Paranasal sinus discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Pharyngeal oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Upper-airway cough syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Complement factor C3 decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 42, 'numAffected': 9}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Complement factor C4 decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Blood uric acid increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Amino acid level increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Blood bicarbonate decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Urobilinogen urine increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Eye swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Ocular hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 70, 'numAffected': 33}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 64, 'numAffected': 30}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 79, 'numAffected': 27}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 42, 'numAffected': 20}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 31, 'numAffected': 13}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 21, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 26, 'numAffected': 13}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Food poisoning', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Tooth abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 188, 'numAffected': 37}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 78, 'numAffected': 30}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 67, 'numAffected': 22}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 27, 'numAffected': 18}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 28, 'numAffected': 13}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Joint stiffness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 20, 'numAffected': 13}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 10}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 10}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 12, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Joint dislocation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Wound', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Folliculitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Vessel puncture site bruise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 9, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Adverse drug reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 10, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Joint injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}], 'seriousEvents': [{'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Abortion induced', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Anaphylactic reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Bacterial infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Bone marrow failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Diarrhoea infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hyponatraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Injection related reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Neuropathy peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Panniculitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Postoperative wound complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Suicide attempt', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 68, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 18.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in the Blood Phenylalanine (Phe) Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1022.4', 'spread': '530.39', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-553.5', 'spread': '652.95', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '53', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-653.3', 'spread': '637.81', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '44', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-672.3', 'spread': '621.06', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 216', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-535.9', 'spread': '573.08', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 240', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-574.9', 'spread': '549.90', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Baseline and Change from Baseline to Week 48, Week 96, Week 144, Week 216, and Week 240', 'description': 'Blood Phe Concentration (Change from Baseline) and Daily Dose in PAL-003 Subjects by Disposition (PAL-003 Population)', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy population consisted of all subjects who received at least one dose of study drug and have post-treatment blood Phe concentration measurements.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics-Plasma Pegvaliase Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'classes': [{'title': 'At Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '961.5', 'spread': '3013.43', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '53', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4127.3', 'spread': '6116.72', 'groupId': 'OG000'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '44', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7457.7', 'spread': '10685.02', 'groupId': 'OG000'}]}]}, {'title': 'Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7196.1', 'spread': '11678.89', 'groupId': 'OG000'}]}]}, {'title': 'Week 216', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7764.4', 'spread': '9292.34', 'groupId': 'OG000'}]}]}, {'title': 'Week 240', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6557.8', 'spread': '9220.95', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Baseline, Week 48, Week 96, Week 144, Week 216 and Week 240', 'description': 'Steady-state Pharmacokinetics (PK) of pegvaliase was measured in subjects who have achieved and maintained target blood Phe', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \\& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \\& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \\& maturation of immune response against pegvaliase, \\& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \\& NCT01560286-165-205 for parent Ph2 demographic data (N=80).'}, {'type': 'SECONDARY', 'title': 'Number of Subjects With Treatment Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'classes': [{'title': 'Subjects with any adverse event', 'categories': [{'measurements': [{'value': '68', 'groupId': 'OG000'}]}]}, {'title': 'Subjects with any serious adverse events', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Subjects with any treatment-related adverse event', 'categories': [{'measurements': [{'value': '65', 'groupId': 'OG000'}]}]}, {'title': 'Any treatment-related serious adverse event', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Up to 109 months.', 'description': 'A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase (PAL-003)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis population consisted of subjects from the study of PAL-003 only'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Positive PEG IgG Antibody', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '29', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '64', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '24', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 104', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 156', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156', 'description': 'The presence of IgG Antibodies against PEG (polyethylene glycol) is measured overtime', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \\& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \\& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \\& maturation of immune response against pegvaliase, \\& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \\& NCT01560286-165-205 for parent Ph2 demographic data (N=80).'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Positive PAL IgG Antibody', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '64', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '63', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '60', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 104', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '48', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 156', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '45', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156', 'description': 'The presence of IgG Antibodies against PAL (phenylalanine ammonia lyase) is measured overtime', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \\& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \\& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \\& maturation of immune response against pegvaliase, \\& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \\& NCT01560286-165-205 for parent Ph2 demographic data (N=80).'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '68'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '46'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '22'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '11'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Withdrawal by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'This was a multicenter study, conducted in 14 study centers of United States.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG 0.001 to a maximum dose of 5.0 mg/kg/week or 375 mg/week by subcutaneous injection'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '28.31', 'spread': '8.66', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '40', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '28', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '67', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'American Indian or Alaska Native', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'White', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '66', 'groupId': 'BG000'}]}]}, {'title': 'Asian', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Weight', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '83.0', 'spread': '26.39', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Body mass index (BMI)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '29.2', 'spread': '8.44', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Total 5 (7.4%) out of 68 subjects BMI are missing due to missing height'}, {'title': 'Baseline blood Phe', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1022.4', 'spread': '530.39', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'All Enrolled Subjects'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-03-31', 'size': 9685378, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-11-03T03:36', 'hasProtocol': True}, {'date': '2016-04-27', 'size': 708516, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-11-03T03:37', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 68}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-01-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'dispFirstSubmitDate': '2019-12-06', 'completionDateStruct': {'date': '2019-01-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-09-15', 'studyFirstSubmitDate': '2009-06-18', 'dispFirstSubmitQcDate': '2019-12-06', 'resultsFirstSubmitDate': '2020-11-03', 'studyFirstSubmitQcDate': '2009-06-18', 'dispFirstPostDateStruct': {'date': '2019-12-10', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2021-10-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-09-15', 'studyFirstPostDateStruct': {'date': '2009-06-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-10-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-01-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in the Blood Phenylalanine (Phe) Concentration', 'timeFrame': 'At Baseline and Change from Baseline to Week 48, Week 96, Week 144, Week 216, and Week 240', 'description': 'Blood Phe Concentration (Change from Baseline) and Daily Dose in PAL-003 Subjects by Disposition (PAL-003 Population)'}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics-Plasma Pegvaliase Concentration', 'timeFrame': 'At Baseline, Week 48, Week 96, Week 144, Week 216 and Week 240', 'description': 'Steady-state Pharmacokinetics (PK) of pegvaliase was measured in subjects who have achieved and maintained target blood Phe'}, {'measure': 'Number of Subjects With Treatment Emergent Adverse Events (TEAEs)', 'timeFrame': 'Up to 109 months.', 'description': 'A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase (PAL-003)'}, {'measure': 'Percentage of Participants With Positive PEG IgG Antibody', 'timeFrame': 'At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156', 'description': 'The presence of IgG Antibodies against PEG (polyethylene glycol) is measured overtime'}, {'measure': 'Percentage of Participants With Positive PAL IgG Antibody', 'timeFrame': 'At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156', 'description': 'The presence of IgG Antibodies against PAL (phenylalanine ammonia lyase) is measured overtime'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Phenylketonuria']}, 'referencesModule': {'references': [{'pmid': '32593547', 'type': 'DERIVED', 'citation': 'Burton BK, Longo N, Vockley J, Grange DK, Harding CO, Decker C, Li M, Lau K, Rosen O, Larimore K, Thomas J; PAL-002 and PAL-004 Investigators. Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up. Mol Genet Metab. 2020 Aug;130(4):239-246. doi: 10.1016/j.ymgme.2020.06.006. Epub 2020 Jun 16.'}, {'pmid': '29973227', 'type': 'DERIVED', 'citation': 'Longo N, Zori R, Wasserstein MP, Vockley J, Burton BK, Decker C, Li M, Lau K, Jiang J, Larimore K, Thomas JA. Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study. Orphanet J Rare Dis. 2018 Jul 4;13(1):108. doi: 10.1186/s13023-018-0858-7.'}]}, 'descriptionModule': {'briefSummary': 'This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.', 'detailedDescription': "PAL-003 is designed to evaluate long-term treatment of subjects who are continuing to take rAvPAL-PEG. Subjects'previous rAvPAL-PEG dosing will continue in PAL-003. In PAL-003, each subject's dose will be adjusted as needed to attain or maintain blood Phe concentrations of 60-600 µmol/L. rAvPAL-PEG dose will be based on either a subject's weight or will be a fixed dose (subjects who have maintained blood Phe levels to 60-600 µmol/L for at least 2 consecutive weeks and who have maintained a stable rAvPAL-PEG dose for at least 2 consecutive weeks). Doses will be evaluated on an individual basis."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '55 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Must have completed participation in previous rAvPAL-PEG studies.\n* Willing and able to provide written, signed informed consent, or, in the case of participants under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained, and prior to any research-related procedures.\n* Willing and able to comply with all study procedures.\n* Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.\n* Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.\n* Maintained a stable diet.\n* In generally good health as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) at Screening.\n\nExclusion Criteria:\n\n* Use of any investigational product (with the exception of rAvPAL-PEG) or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.\n* Use of any medication that is intended to treat PKU within 14 days prior to the administration of study drug.\n* Use or planned use of any injectable drugs containing PEG (other than rAvPAL-PEG), including Depo-Provera during study participation.\n* A prior reaction that included systemic symptoms (eg, generalized hives, respiratory or gastrointestinal problems, hypotension, angioedema, anaphylaxis) to rAvPAL-PEG or a PEG-containing product.\n* Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study.Concurrent disease or condition that would interfere with study participation or safety (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease).\n* Any condition that, in the view of the PI, places the subject at high risk of poor treatment compliance or of not completing the study.\n* Known hypersensitivity to rAvPAL-PEG or its excipients, including hypersensitivity reactions that necessitated early termination from previous rAvPAL-PEG studies.\n* Alanine aminotransferase (ALT) concentration \\> 2 times the upper limit of normal.\n* Creatinine \\> 1.5 times the upper limit of normal.'}, 'identificationModule': {'nctId': 'NCT00924703', 'briefTitle': 'Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKU', 'orgStudyIdInfo': {'id': 'PAL-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'rAvPAL-PEG', 'description': 'rAvPAL-PEG in varying doses dependent on safety and efficacy.', 'interventionNames': ['Drug: rAvPAL-PEG']}], 'interventions': [{'name': 'rAvPAL-PEG', 'type': 'DRUG', 'otherNames': ['Pegvaliase'], 'description': "The doses are planned to be in the same range as those tested in PAL-002 or PAL-004 and then modified either by increasing or decreasing the dose, adhering to an upper limit up to 5.0 mg/kg per week or 375 mg/week, considering each subject's individual responses related to safety and efficacy.", 'armGroupLabels': ['rAvPAL-PEG']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "The Children's Hospital", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann and Robert H Lurie Children's Hospital", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Louisville, Kosair Charities Pediatric Clinical Research Unit', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '65212', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University Center for Applied Research Sciences', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '68198', 'city': 'Omaha', 'state': 'Nebraska', 'country': 'United States', 'facility': 'Nebraska Medical Center', 'geoPoint': {'lat': 41.25626, 'lon': -95.94043}}, {'zip': '12208', 'city': 'Albany', 'state': 'New York', 'country': 'United States', 'facility': 'Albany Medical Center', 'geoPoint': {'lat': 42.65258, 'lon': -73.75623}}, {'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': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health and Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospital', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Medical College of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}], 'overallOfficials': [{'name': 'Medical Director, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'BioMarin Pharmaceutical'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}