Ignite Creation Date:
2025-12-25 @ 4:23 AM
Ignite Modification Date:
2025-12-26 @ 3:25 AM
Study NCT ID:
NCT00007020
Status:
COMPLETED
Last Update Posted:
2023-10-03
First Post:
2000-12-06
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D052919', 'term': 'Refsum Disease, Infantile'}, {'id': 'D015211', 'term': 'Zellweger Syndrome'}, {'id': 'D000326', 'term': 'Adrenoleukodystrophy'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D002779', 'term': 'Cholestasis'}], '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': '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': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D000309', 'term': 'Adrenal Insufficiency'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D002793', 'term': 'Cholic Acids'}, {'id': 'D019826', 'term': 'Cholic Acid'}], 'ancestors': [{'id': 'D001647', 'term': 'Bile Acids and Salts'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D002757', 'term': 'Cholanes'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@retrophin.com', 'phone': '+1 8776595', 'title': 'Retrophin Medical Information', 'phoneExt': '518', 'organization': 'Retrophin, Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'CAC-91-10-10 includes data from patients enrolled from 1992-2009. The study end date was arbitrarily chosen as data cut-off point; the disease requires life-long treatment, no final study visit was done. Many patients continued onto study CAC-002-01'}}, 'adverseEventsModule': {'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'eventGroups': [{'id': 'EG000', 'title': 'Cholic Acid', 'description': 'All patients entered into the study and treated with Cholic Acid. 85 patients enrolled in the study and 79 received treatment (safety set).', 'otherNumAtRisk': 79, 'deathsNumAtRisk': 79, 'otherNumAffected': 35, 'seriousNumAtRisk': 79, 'deathsNumAffected': 13, 'seriousNumAffected': 20}], 'otherEvents': [{'term': 'Disease progression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Injection site inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hallucination', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Ovarian cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Ovarian failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Amino acid level increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Occult blood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Apnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pulmonary congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hypotonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Anal fistula', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Ascites', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Enteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Inguinal hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cholestasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Jaundice', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Portal hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dermatitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Scoliosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Iron deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Malnutrition', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vitamin E deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Bronchopneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'seriousEvents': [{'term': 'Nutritional condition abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Respiratory disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Respiratory distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Coagulopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Disease progression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastric ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumoperitoneum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Jaundice', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Rotavirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 79, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Excretion of Atypical Bile Acids in Urine by Category', 'denoms': [{'units': 'Participants', 'counts': [{'value': '70', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'Patients treated'}], 'classes': [{'title': 'Baseline, no atypical bile acid excretion', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, no atypical bile acid excretion', 'categories': [{'measurements': [{'value': '51', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, slight atypical bile acid excretion', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, slight atypical bile acid excretion', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, significant atypical bile acid excretion', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'OT, significant atypical bile acid excretion', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, marked atypical bile acid excretion', 'categories': [{'measurements': [{'value': '33', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, marked atypical bile acid excretion', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment'}, {'type': 'SECONDARY', 'title': 'Change in Liver Function Tests (LFTs) Measured in Serum', 'denoms': [{'units': 'Participants', 'counts': [{'value': '70', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'Patients treated'}], 'classes': [{'title': 'Baseline, ALT <ULN', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, ALT <ULN', 'categories': [{'measurements': [{'value': '49', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, ULN ≤ ALT <2 x ULN', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, ULN ≤ ALT <2 x ULN', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, 2 ULN ≤ ALT <3 x ULN', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, 2 ULN ≤ ALT <3 x ULN', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, ALT ≥3 x ULN', 'categories': [{'measurements': [{'value': '29', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, ALT ≥3 x ULN', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, AST <ULN', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, AST <ULN', 'categories': [{'measurements': [{'value': '38', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, ULN ≤ AST <2 x ULN', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, ULN ≤ AST <2 x ULN', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, 2 ULN ≤ AST <3 x ULN', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, 2 ULN ≤ AST <3 x ULN', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, AST ≥3 x ULN', 'categories': [{'measurements': [{'value': '35', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, AST ≥3 x ULN', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Patients with elevations of liver function tests (alanine transaminase \\[ALT\\], aspartate transaminase \\[AST\\]) measured as multiples of the upper limit of normal (ULN) at baseline (worst value) and on treatment (best value)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients treated and with at least 1 pre-treatment and 1 post-Treatment assessment'}, {'type': 'SECONDARY', 'title': 'Liver Histology', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'All patients entered into the study'}], 'classes': [{'title': 'Periportal inflammation at BL, qualitative', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Periportal inflammation OT, qualitative', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'Lobular inflammation at BL, qualitative', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Lobular inflammation OT, qualitative', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Inflammation (not spec.) at BL, qualitative', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Inflammation (not spec.) OT, qualitative', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Bridging fibrosis at BL, qualitative', 'categories': [{'measurements': [{'value': '25', 'groupId': 'OG000'}]}]}, {'title': 'Bridging fibrosis OT, qualitative', 'categories': [{'measurements': [{'value': '31', 'groupId': 'OG000'}]}]}, {'title': 'Fibrosis (not specified) at BL, qualitative', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Fibrosis (not specified) OT, qualitative', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Cholestasis at BL, qualitative', 'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}]}]}, {'title': 'Cholestasis OT, qualitative', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Giant cells at BL, qualitative', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Giant cells OT, qualitative', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Necrosis at BL, qualitative', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'Necrosis OT, qualitative', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Periportal inflammation at BL, quantitative', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Periportal inflammation OT, quantitative', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Lobular inflammation at BL, quantitative', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Lobular inflammation OT, quantitative', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Inflammation (not specified) at BL, quantitative', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Inflammation (not specified) OT, quantitative', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Bridging fibrosis at baseline, quantitative', 'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000'}]}]}, {'title': 'Bridging fibrosis OT, quantitative', 'categories': [{'measurements': [{'value': '27', 'groupId': 'OG000'}]}]}, {'title': 'Fibrosis (not specified) at BL, quantitative', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Fibrosis (not specified) OT, quantitative', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Cholestasis at BL, quantitative', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Cholestasis OT, quantitative', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Giant cells at BL, quantitative', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Giant cells OT, quantitative', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Necrosis at BL, quantitative', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Necrosis OT, quantitative', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At baseline (if no historical data were available) and between 1 and 6 months following treatment start.', 'description': 'Patients (number, percentage) with pathological findings for qualitative (the presence of inflammation, fibrosis, necrosis, giant cells and cholestasis) and quantitative (the degrees of the aforementioned histologic features) liver histopathology at baseline (BL) and on treatment (OT).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All patients entered into the study'}, {'type': 'SECONDARY', 'title': 'Height and Weight', 'denoms': [{'units': 'Participants', 'counts': [{'value': '70', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'Patients treated'}], 'classes': [{'title': 'Height percentile, baseline', 'categories': [{'measurements': [{'value': '30.1', 'spread': '5.5', 'groupId': 'OG000'}]}]}, {'title': 'Height percentile, on treatment', 'categories': [{'measurements': [{'value': '44.0', 'spread': '6.1', 'groupId': 'OG000'}]}]}, {'title': 'Weight percentile, baseline', 'categories': [{'measurements': [{'value': '21.3', 'spread': '3.7', 'groupId': 'OG000'}]}]}, {'title': 'Weight percentile, on treatment', 'categories': [{'measurements': [{'value': '42.3', 'spread': '4.8', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Change in height/weight percentiles from baseline (worst value) to the best on-treatment value, based on CDC (Centres for Disease Control and Prevention, US) growth chart percentiles', 'unitOfMeasure': 'Percentile', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment'}, {'type': 'SECONDARY', 'title': 'Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'All patients entered into the study and treated'}], 'classes': [{'title': 'Patients at risk', 'categories': [{'measurements': [{'value': '79', 'groupId': 'OG000'}]}]}, {'title': 'Patients with any AE', 'categories': [{'measurements': [{'value': '38', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Number of patients with any adverse event', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients entered into the study and treated'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Change in Bilirubin Measured in Serum', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '689', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cholic Acid', 'description': 'All patients entered into the study'}], 'classes': [{'title': 'Baseline, bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '0.5', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, direct bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '82', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.2', 'spread': '5.6', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, direct bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '44', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '195', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '1.8', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, indirect bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '0.9', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, indirect bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.2', 'groupId': 'OG000'}]}]}, {'title': 'Baseline, total bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '86', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.8', 'spread': '10.6', 'groupId': 'OG000'}]}]}, {'title': 'On treatment, total bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}, {'units': 'Number of Test Results', 'counts': [{'value': '288', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.5', 'spread': '5.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and on treatment (every 1, 3, or 6 months, depending on protocol version, for an average of 2.8 years)', 'description': 'Bilirubin concentration in serum at baseline and on treatment', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Number of Test Results', 'denomUnitsSelected': 'Number of Test Results', 'populationDescription': 'All available bilirubin laboratory data are collected then grouped by bilirubin test type and collection time points (baseline vs. on treatment). Not all participant had available data, and each participant may have more than one type of bilirubin collected at a time point, and may have multiple on-treatment collections.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cholic Acid', 'description': 'Cholic acid capsules, each containing 250 mg of cholic acid, or a liquid preparation of 15 mg/mL cholic acid, to be administered orally in a dose of 15 mg/kg body weight/day.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '85'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '41'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '16'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'Pt withdrawn (worsening cholestasis)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Pt withdrawn (liver transplant)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'Pt cared for by outside physician', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'No evidence of treatment', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}]}], 'recruitmentDetails': 'A total of 85 patients were enrolled.', 'preAssignmentDetails': "Of the patients diagnosed with a defect in bile acid synthesis during routine diagnostic procedures at the Cincinnati Children's Hospital Medical Center (CCHMC) Mass Spectrometry Laboratory, 85 patients were invited to participate in the study."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Cholic Acid', 'description': 'All patients entered into the study'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '77', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '3', 'spread': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Continuous age data were not available for 8 out of 85 patients.'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '31', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '50', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Information on gender was not recorded for 4 patients.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '85', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 85}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1992-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'dispFirstSubmitDate': '2013-05-21', 'completionDateStruct': {'date': '2009-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-29', 'studyFirstSubmitDate': '2000-12-06', 'dispFirstSubmitQcDate': '2013-05-21', 'resultsFirstSubmitDate': '2019-04-12', 'studyFirstSubmitQcDate': '2000-12-06', 'dispFirstPostDateStruct': {'date': '2013-05-29', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2023-10-03', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-06-29', 'studyFirstPostDateStruct': {'date': '2000-12-07', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-07-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2009-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in Bilirubin Measured in Serum', 'timeFrame': 'Baseline and on treatment (every 1, 3, or 6 months, depending on protocol version, for an average of 2.8 years)', 'description': 'Bilirubin concentration in serum at baseline and on treatment'}], 'primaryOutcomes': [{'measure': 'Number of Participants With Excretion of Atypical Bile Acids in Urine by Category', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)'}], 'secondaryOutcomes': [{'measure': 'Change in Liver Function Tests (LFTs) Measured in Serum', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Patients with elevations of liver function tests (alanine transaminase \\[ALT\\], aspartate transaminase \\[AST\\]) measured as multiples of the upper limit of normal (ULN) at baseline (worst value) and on treatment (best value)'}, {'measure': 'Liver Histology', 'timeFrame': 'At baseline (if no historical data were available) and between 1 and 6 months following treatment start.', 'description': 'Patients (number, percentage) with pathological findings for qualitative (the presence of inflammation, fibrosis, necrosis, giant cells and cholestasis) and quantitative (the degrees of the aforementioned histologic features) liver histopathology at baseline (BL) and on treatment (OT).'}, {'measure': 'Height and Weight', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Change in height/weight percentiles from baseline (worst value) to the best on-treatment value, based on CDC (Centres for Disease Control and Prevention, US) growth chart percentiles'}, {'measure': 'Adverse Events', 'timeFrame': 'Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years', 'description': 'Number of patients with any adverse event'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ["Infantile Refsum's Disease", 'Zellweger Syndrome', 'Adrenoleukodystrophy', 'Peroxisomal Disorders', 'Cholestasis']}, 'referencesModule': {'references': [{'pmid': '28644367', 'type': 'DERIVED', 'citation': 'Heubi JE, Bove KE, Setchell KDR. Oral Cholic Acid Is Efficacious and Well Tolerated in Patients With Bile Acid Synthesis and Zellweger Spectrum Disorders. J Pediatr Gastroenterol Nutr. 2017 Sep;65(3):321-326. doi: 10.1097/MPG.0000000000001657.'}]}, 'descriptionModule': {'briefSummary': 'OBJECTIVES:\n\nI. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism\n\nII. To assess the safety and tolerability of cholic acid', 'detailedDescription': "Investigational Plan:\n\nA Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism.\n\nThe study was begun with a single study site at Cincinnati Children's Hospital Medical Center (CCHMC), but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the center's screening/diagnostic program.\n\nPatients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study.\n\nThe primary interventions for the study were:\n\n1. Administration of study drug.\n2. Collection of baseline physical exam, vital signs, blood and urine samples for laboratory tests.\n3. Collection of periodic physical exam, vital signs, blood and urine samples for laboratory tests during the period of administration of the study drug.\n4. Collection of any adverse event information.\n\nTime and Events Schedule:\n\nBaseline:\n\n1. Confirm eligibility\n2. Obtain written informed consent from patient and/or parents/legal guardian\n3. Collect demographic data and disease and medication history, including family history\n\n Baseline and Ongoing:\n4. Obtain body weight\n5. Record adverse events\n6. Obtain blood and urine samples for laboratory tests\n7. Initiate study drug therapy \\& monitor study drug therapy and adjust dose as needed"}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics--\n\nClinical or biochemical evidence of liver disease, unexplained fat-soluble vitamin malabsorption, or peroxisomal dysfunction that compromises bile acid biosynthesis\n\nInclusion criteria for enrollment were:\n\n* Infants \\< age 3 months\n* Children presenting for evaluation of cholestasis defined as a conjugated bilirubin \\> 2mg/dl or increased serum bile acids\n* Older subjects of any age with cholestatic liver disease if urine screens suggested that they had inborn errors of bile acid metabolism\n* Confirmation of a diagnosis of an inborn error of bile acid synthesis based upon urine analysis by FAB-MS to determine whether specific abnormalities in bile acid synthesis are indicated\n* The patient and/or parent/legal guardian must have signed the written informed consent document before study start.\n* The patient must be willing and able to comply with all study assessments and procedures.'}, 'identificationModule': {'nctId': 'NCT00007020', 'briefTitle': 'Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism', 'orgStudyIdInfo': {'id': 'CAC-91-10-10'}, 'secondaryIdInfos': [{'id': 'CCHMC-91-10-10', 'type': 'OTHER', 'domain': "Cincinnati Children's Hospital Medical Center"}, {'id': 'NCRR-M01RR08084-0009', 'type': 'OTHER', 'domain': 'National Center For Research Resources (NCRR)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Cholic Acid', 'interventionNames': ['Drug: Cholic Acids']}], 'interventions': [{'name': 'Cholic Acids', 'type': 'DRUG', 'otherNames': ['Cholic', 'Cholic Acid', 'Cholic Acid Capsules'], 'description': '10-15 mg/kg body weight/day taken orally.', 'armGroupLabels': ['Cholic Acid']}]}, 'contactsLocationsModule': {'locations': [{'zip': '45229-3039', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}], 'overallOfficials': [{'name': 'James Heubi, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital Medical Center, Cincinnati"}, {'name': 'Kenneth Setchell, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital Medical Center, Cincinnati"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': "Children's Hospital Medical Center, Cincinnati", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}