Viewing Study NCT04674761

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Ignite Creation Date: 2025-12-24 @ 1:05 PM
Ignite Modification Date: 2025-12-28 @ 9:12 PM
Study NCT ID: NCT04674761
Status: COMPLETED
Last Update Posted: 2023-11-02
First Post: 2020-12-10
Is NOT Gene Therapy: True
Has Adverse Events: True
Brief Title: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-10-04', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D016738', 'term': 'Alagille Syndrome'}], 'ancestors': [{'id': 'D002780', 'term': 'Cholestasis, Intrahepatic'}, {'id': 'D002779', 'term': 'Cholestasis'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000713258', 'term': 'odevixibat'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@albireopharma.com', 'phone': 'medinfo@albireopharma.com', 'title': 'Albireo AB', 'organization': 'Albireo AB'}, 'certainAgreement': {'otherDetails': "Albireo will retain the ownership of all data. All proposed publications based on this study must be subject to the sponsor's approval requirements.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.', 'eventGroups': [{'id': 'EG000', 'title': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks.\n\nOdevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.', 'otherNumAtRisk': 35, 'deathsNumAtRisk': 35, 'otherNumAffected': 26, 'seriousNumAtRisk': 35, 'deathsNumAffected': 0, 'seriousNumAffected': 5}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.\n\nPlacebo: Placebo identical in appearance to experimental drug (odevixibat).', 'otherNumAtRisk': 17, 'deathsNumAtRisk': 17, 'otherNumAffected': 12, 'seriousNumAtRisk': 17, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Anaemia macrocytic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Coagulopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Cataract cortical', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Aphthous ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 11, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Faeces discoloured', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Faeces soft', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Frequent bowel movements', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 8, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Jaundice', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 4, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Otitis externa', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Subcutaneous abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Viral rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Vaccination complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Blood pressure diastolic increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Blood triglycerides increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'International normalised ratio increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Platelet count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Vitamin A decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Vitamin E decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Hypophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': "Bell's palsy", 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Nasal turbinate hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pharyngeal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Rhinitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Skin lesion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}], 'seriousEvents': [{'term': 'International normalised ratio increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Cerumen impaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Otitis media chronic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Rhinovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Subcutaneous abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Adenoidal hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 35, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Scratching Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks.\n\nOdevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.\n\nPlacebo: Placebo identical in appearance to experimental drug (odevixibat).'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.69', 'spread': '0.174', 'groupId': 'OG000'}, {'value': '-0.8', 'spread': '0.233', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0012', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.88', 'ciLowerLimit': '-1.44', 'ciUpperLimit': '-0.33', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.277', 'groupDescription': 'The analysis is based on a mixed model for repeated measures (MMRM) using a restricted maximum likelihood (REML) with baseline score as a covariate, and baseline age stratification, baseline direct bilirubin, treatment group, time (in months), and treatment-by-time interaction as fixed effects. One-sided p-value was reported.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.', 'description': 'Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Serum Bile Acid Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks.\n\nOdevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.\n\nPlacebo: Placebo identical in appearance to experimental drug (odevixibat).'}], 'classes': [{'categories': [{'measurements': [{'value': '-90.35', 'spread': '21.336', 'groupId': 'OG000'}, {'value': '22.39', 'spread': '28.463', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0006', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-112.74', 'ciLowerLimit': '-178.78', 'ciUpperLimit': '-46.69', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '32.864', 'groupDescription': 'The analysis is based on a mixed model for repeated measures (MMRM) using a restricted maximum likelihood (REML) with baseline serum bile acid (sBA) concentration data as a covariate, and baseline age stratification, treatment group, visits (Weeks 4, 8, 12, 16, 20, 24), and treatment-by-visit interaction as fixed effects. The comparison of treatment difference in change from baseline to the average of Week 20 and Week 24 is estimated and tested using contrast. One-sided p-value was reported.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.', 'description': 'Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24', 'unitOfMeasure': 'μmol/L', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks (120 μg/kg/day).\n\nOdevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.\n\nPlacebo: Placebo identical in appearance to experimental drug (odevixibat).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '52', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks (120 μg/kg/day).\n\nOdevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.\n\nPlacebo: Placebo identical in appearance to experimental drug (odevixibat).'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '35', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '52', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '6.73', 'spread': '3.780', 'groupId': 'BG000'}, {'value': '5.40', 'spread': '4.411', 'groupId': 'BG001'}, {'value': '6.29', 'spread': '4.003', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'categories': [{'title': '< 10 years', 'measurements': [{'value': '29', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '42', 'groupId': 'BG002'}]}, {'title': '> = 10 years and < 18 years', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'White', 'measurements': [{'value': '30', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '43', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Other', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Malaysia', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}, {'title': 'Netherlands', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'Turkey', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Belgium', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Poland', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Genetic Mutation', 'classes': [{'categories': [{'title': 'JAG1', 'measurements': [{'value': '32', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '48', 'groupId': 'BG002'}]}, {'title': 'NOTCH2', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-08-28', 'size': 23238419, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-09-07T13:32', 'hasProtocol': True}, {'date': '2022-09-27', 'size': 1497969, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-09-07T13:33', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 52}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-03-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-10', 'completionDateStruct': {'date': '2022-09-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-10-10', 'studyFirstSubmitDate': '2020-12-10', 'resultsFirstSubmitDate': '2023-09-08', 'studyFirstSubmitQcDate': '2020-12-17', 'lastUpdatePostDateStruct': {'date': '2023-11-02', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-10-10', 'studyFirstPostDateStruct': {'date': '2020-12-19', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-11-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-09-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Scratching Score', 'timeFrame': 'Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.', 'description': 'Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.'}], 'secondaryOutcomes': [{'measure': 'Serum Bile Acid Levels', 'timeFrame': 'Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.', 'description': 'Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ALGS', 'Alagille syndrome'], 'conditions': ['Alagille Syndrome']}, 'referencesModule': {'references': [{'pmid': '38670135', 'type': 'DERIVED', 'citation': 'Ovchinsky N, Aumar M, Baker A, Baumann U, Bufler P, Cananzi M, Czubkowski P, Durmaz O, Fischer R, Indolfi G, Karnsakul WW, Lacaille F, Lee WS, Maggiore G, Rosenthal P, Ruiz M, Sokal E, Sturm E, van der Woerd W, Verkade HJ, Wehrman A, Clemson C, Yu Q, Ni Q, Ruvido J, Manganaro S, Mattsson JP. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.'}]}, 'descriptionModule': {'briefSummary': 'Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.', 'detailedDescription': 'Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Genetically confirmed diagnosis of Alagille syndrome\n* History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument\n* Elevated serum bile acid level\n\nKey Exclusion Criteria:\n\n* History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)\n* History of liver transplant, or a liver transplant is planned within 6 months of randomization\n* ALT \\>10× upper limit of normal (ULN) at screening\n* Total bilirubin \\>15 × ULN at screening\n* Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome'}, 'identificationModule': {'nctId': 'NCT04674761', 'acronym': 'ASSERT', 'briefTitle': 'Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ipsen'}, 'officialTitle': 'A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)', 'orgStudyIdInfo': {'id': 'A4250-012'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 24 weeks.', 'interventionNames': ['Drug: Odevixibat']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Capsules for oral administration (to match active) once daily for 24 weeks.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Odevixibat', 'type': 'DRUG', 'otherNames': ['A4250'], 'description': 'Odevixibat is a small molecule and selective inhibitor of IBAT.', 'armGroupLabels': ['Odevixibat (A4250)']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo identical in appearance to experimental drug (odevixibat).', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California - 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