Ignite Creation Date:
2025-12-25 @ 2:03 AM
Ignite Modification Date:
2026-03-05 @ 12:30 AM
Study NCT ID:
NCT01903460
Status:
COMPLETED
Last Update Posted:
2019-03-28
First Post:
2013-07-16
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@mirumpharma.com', 'phone': '+1 650-667-4085', 'title': 'Study Physician', 'organization': 'Mirum'}, 'certainAgreement': {'otherDetails': 'If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.', 'otherNumAtRisk': 6, 'otherNumAffected': 6, 'seriousNumAtRisk': 6, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.', 'otherNumAtRisk': 8, 'otherNumAffected': 7, 'seriousNumAtRisk': 8, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Placebo Overall', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.', 'otherNumAtRisk': 6, 'otherNumAffected': 4, 'seriousNumAtRisk': 6, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Deafness unilateral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Middle ear inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 8, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Abdominal pain lower', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 9, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Frequent bowel movements', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Proctalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Crying', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Feeling abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 6, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Viral rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Anal injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Excoriation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Body temperature increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Vitamin E deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Abnormal behaviour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Drug eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Rash generalised', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}, {'term': 'Rash maculo-papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG002', 'title': 'LUM001 Overall', 'description': 'Participants received either dose of LUM001 for up to 10 or 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'OG003', 'title': 'Placebo Overall', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}], 'classes': [{'categories': [{'measurements': [{'value': '-82.864', 'spread': '50.1513', 'groupId': 'OG000'}, {'value': '-49.388', 'spread': '43.4732', 'groupId': 'OG001'}, {'value': '-66.126', 'spread': '33.1208', 'groupId': 'OG002'}, {'value': '-42.157', 'spread': '50.0903', 'groupId': 'OG003'}]}]}], 'analyses': [{'pValue': '0.5740', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-40.707', 'ciLowerLimit': '-191.679', 'ciUpperLimit': '110.265', 'groupDescription': 'Analysis of LUM001 140ug/kg/day', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.9147', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-7.231', 'ciLowerLimit': '-148.726', 'ciUpperLimit': '134.264', 'groupDescription': 'Analysis of LUM001 280ug/kg/day', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.6954', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-23.969', 'ciLowerLimit': '-151.969', 'ciUpperLimit': '104.031', 'groupDescription': 'Analysis of all doses of LUM001', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.', 'unitOfMeasure': 'umol/L', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intent-to-Treat (mITT) population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG002', 'title': 'LUM001 Overall', 'description': 'Participants received either dose of LUM001 for up to 10 or 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'OG003', 'title': 'Placebo Overall', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}], 'classes': [{'title': 'ALT', 'categories': [{'measurements': [{'value': '59.3', 'spread': '20.99', 'groupId': 'OG000'}, {'value': '10.5', 'spread': '18.06', 'groupId': 'OG001'}, {'value': '34.9', 'spread': '13.86', 'groupId': 'OG002'}, {'value': '2.7', 'spread': '21.06', 'groupId': 'OG003'}]}]}, {'title': 'AST', 'categories': [{'measurements': [{'value': '37.2', 'spread': '13.64', 'groupId': 'OG000'}, {'value': '-2.7', 'spread': '11.70', 'groupId': 'OG001'}, {'value': '17.3', 'spread': '8.98', 'groupId': 'OG002'}, {'value': '13.2', 'spread': '13.63', 'groupId': 'OG003'}]}]}, {'title': 'ALP', 'categories': [{'measurements': [{'value': '71.4', 'spread': '53.35', 'groupId': 'OG000'}, {'value': '31.6', 'spread': '43.59', 'groupId': 'OG001'}, {'value': '51.5', 'spread': '36.13', 'groupId': 'OG002'}, {'value': '19.7', 'spread': '60.76', 'groupId': 'OG003'}]}]}], 'analyses': [{'pValue': '0.0783', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '56.7', 'ciLowerLimit': '-7.2', 'ciUpperLimit': '120.6', 'groupDescription': 'Analysis of LUM001 140ug/kg/day for ALT', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.7827', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '7.8', 'ciLowerLimit': '-51.4', 'ciUpperLimit': '67.0', 'groupDescription': 'Analysis of LUM001 280ug/kg/day for ALT', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.2235', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '32.2', 'ciLowerLimit': '-21.9', 'ciUpperLimit': '86.3', 'groupDescription': 'Analysis of all doses of LUM001 for ALT', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.2372', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '24.0', 'ciLowerLimit': '-17.5', 'ciUpperLimit': '65.5', 'groupDescription': 'Analysis of LUM001 140ug/kg/day for AST', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.3914', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-15.8', 'ciLowerLimit': '-54.1', 'ciUpperLimit': '22.4', 'groupDescription': 'Analysis of LUM001 280ug/kg/day for AST', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.8081', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.1', 'ciLowerLimit': '-31.0', 'ciUpperLimit': '39.1', 'groupDescription': 'Analysis of all doses of LUM001 for AST', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.5748', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '51.7', 'ciLowerLimit': '-140.3', 'ciUpperLimit': '243.7', 'groupDescription': 'Analysis of LUM001 140ug/kg/day for ALP', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.8835', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '11.9', 'ciLowerLimit': '-158.4', 'ciUpperLimit': '182.2', 'groupDescription': 'Analysis of LUM001 280ug/kg/day for ALP', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.6917', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '31.8', 'ciLowerLimit': '-135.8', 'ciUpperLimit': '199.4', 'groupDescription': 'Analysis of all doses of LUM001 for ALP', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The mITT population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '14', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'OG002', 'title': 'LUM001 Overall', 'description': 'Participants received either dose of LUM001 for up to 10 or 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'OG003', 'title': 'Placebo Overall', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}], 'classes': [{'title': 'Patient ItchRO', 'categories': [{'measurements': [{'value': '-1.159', 'spread': '0.5396', 'groupId': 'OG000'}, {'value': '-0.608', 'spread': '0.4399', 'groupId': 'OG001'}, {'value': '-0.883', 'spread': '0.3484', 'groupId': 'OG002'}, {'value': '-0.811', 'spread': '0.5684', 'groupId': 'OG003'}]}]}, {'title': 'Observer ItchRO', 'categories': [{'measurements': [{'value': '-0.802', 'spread': '0.2732', 'groupId': 'OG000'}, {'value': '-0.419', 'spread': '0.2318', 'groupId': 'OG001'}, {'value': '-0.610', 'spread': '0.1776', 'groupId': 'OG002'}, {'value': '-0.592', 'spread': '0.2690', 'groupId': 'OG003'}]}]}], 'analyses': [{'pValue': '0.6907', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.348', 'ciLowerLimit': '-2.468', 'ciUpperLimit': '1.772', 'groupDescription': 'Analysis of LUM001 140ug/kg/day for Patient ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.7897', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.202', 'ciLowerLimit': '-1.647', 'ciUpperLimit': '2.052', 'groupDescription': 'Analysis of LUM001 280ug/kg/day for Patient ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.9203', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.073', 'ciLowerLimit': '-1.850', 'ciUpperLimit': '1.705', 'groupDescription': 'Analysis of all doses of LUM001 for Patient ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.5966', 'groupIds': ['OG000', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.210', 'ciLowerLimit': '-1.038', 'ciUpperLimit': '0.618', 'groupDescription': 'Analysis of LUM001 140ug/kg/day for Observer ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.6320', 'groupIds': ['OG001', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.173', 'ciLowerLimit': '-0.580', 'ciUpperLimit': '0.926', 'groupDescription': 'Analysis of LUM001 280ug/kg/day for Observer ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.9547', 'groupIds': ['OG002', 'OG003'], 'paramType': 'LS mean difference from placebo', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.019', 'ciLowerLimit': '-0.710', 'ciUpperLimit': '0.673', 'groupDescription': 'Analysis of all doses of LUM001 for Observer ItchRO', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The mITT population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'FG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'FG002', 'title': 'Placebo Cohort A', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'FG003', 'title': 'Placebo Cohort B', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}, {'value': '20', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'LUM001 140ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 140ug/kg/day, then received 8 to 10 weeks of treatment at either 140ug/kg/day or the highest tolerated dose below 140ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'BG001', 'title': 'LUM001 280ug/kg/Day', 'description': 'Participants received an escalating dose of LUM001 over 3 to 5 weeks, from 14ug/kg/day to 280ug/kg/day, then received 8 to 10 weeks of treatment at either 280ug/kg/day or the highest tolerated dose below 280ug/kg/day. Participants were then followed for 4 weeks after treatment.'}, {'id': 'BG002', 'title': 'Placebo Cohort A', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'BG003', 'title': 'Placebo Cohort B', 'description': 'Participants received LUM001-matching placebo for up to 13 weeks, then were followed for 4 weeks after treatment.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '5.8', 'spread': '4.49', 'groupId': 'BG000'}, {'value': '6.8', 'spread': '6.73', 'groupId': 'BG001'}, {'value': '5.0', 'spread': '2.00', 'groupId': 'BG002'}, {'value': '4.3', 'spread': '3.21', 'groupId': 'BG003'}, {'value': '5.9', 'spread': '4.93', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': '< 2 years', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}]}]}, {'title': '2 to 4 years', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}, {'value': '7', 'groupId': 'BG004'}]}]}, {'title': '5 to 8 years', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '5', 'groupId': 'BG004'}]}]}, {'title': '9 to 12 years', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}]}]}, {'title': '13 to 18 years', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}, {'value': '10', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '10', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}, {'value': '20', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'All randomized participants'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 20}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2015-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-15', 'studyFirstSubmitDate': '2013-07-16', 'resultsFirstSubmitDate': '2015-11-02', 'studyFirstSubmitQcDate': '2013-07-16', 'lastUpdatePostDateStruct': {'date': '2019-03-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-11-02', 'studyFirstPostDateStruct': {'date': '2013-07-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-12-07', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.'}, {'measure': 'Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores', 'timeFrame': 'Baseline to 13 weeks or end of treatment', 'description': 'The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Alagille Syndrome']}, 'descriptionModule': {'briefSummary': 'The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '12 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Diagnosis of Alagille Syndrome\n2. Evidence of cholestasis\n3. Moderate to severe pruritus\n4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures\n\nExclusion Criteria:\n\n1. Surgical disruption of the enterohepatic circulation\n2. Liver transplant\n3. History or presence of other concomitant liver disease\n4. Females who are pregnant or lactating\n5. Known HIV infection'}, 'identificationModule': {'nctId': 'NCT01903460', 'acronym': 'IMAGO', 'briefTitle': 'Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Paediatric Patients With Alagille Syndrome', 'orgStudyIdInfo': {'id': 'LUM001-302'}, 'secondaryIdInfos': [{'id': '2012-005346-38', 'type': 'EUDRACT_NUMBER'}, {'id': 'SHP625-302', 'type': 'OTHER', 'domain': 'Shire Development LLC'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'LUM001', 'description': 'LUM001 administered orally once each day', 'interventionNames': ['Drug: LUM001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo administered orally once each day', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'LUM001', 'type': 'DRUG', 'armGroupLabels': ['LUM001']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'B4 6NH', 'city': 'Birmingham', 'state': 'West Midlands', 'country': 'United Kingdom', 'facility': "Birmingham Children's Hospital", 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'LS9 7TF', 'city': 'Leeds', 'state': 'West Yorkshire', 'country': 'United Kingdom', 'facility': 'Leeds Teaching Hospitals', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'zip': 'SE5 9RS', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Kings College Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Mirum'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}