Ignite Creation Date:
2025-12-24 @ 11:47 AM
Ignite Modification Date:
2025-12-29 @ 6:09 AM
Study NCT ID:
NCT04718961
Status:
TERMINATED
Last Update Posted:
2024-08-06
First Post:
2021-01-19
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-12-21', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'C535932', 'term': 'Intrahepatic Cholestasis of Pregnancy'}, {'id': 'D011537', 'term': 'Pruritus'}, {'id': 'D002779', 'term': 'Cholestasis'}], 'ancestors': [{'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012877', 'term': 'Skin Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000627853', 'term': 'volixibat'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@mirumpharma.com', 'phone': '650-667-4085', 'title': 'Mirum Clinical Trials', 'organization': 'Mirum Pharmaceuticals'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks**', 'description': "Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.", 'eventGroups': [{'id': 'EG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Apnoic Episodes', 'notes': 'Newborn AE', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Heart Burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intermittent Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intermittent Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intermittent Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Postnatal Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Premature Labor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Raised Blood Pressure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Threated Pre-Term Labor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0}, {'groupId': 'EG003', 'numAtRisk': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Assess the Safety and Tolerability of Volixibat in Participants With ICP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Through to end of treatment, up to 21 weeks', 'description': 'To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints:\n\nProportion of participants experiencing one or more of the following:\n\nTreatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs.\n\nClinically significant laboratory abnormalities', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': "Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility."}, {'type': 'SECONDARY', 'title': 'Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.5', 'spread': '2.40', 'groupId': 'OG000'}, {'value': '1.3', 'spread': '2.31', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Through to end of treatment, up to 21 weeks', 'description': 'Adult Itch Reported Outcome (ItchRO) is a 0 to 10 scale with 0 being "no itch" and 10 being "worst possible itch" where participants are responding to the following question "How would you rate the worst itch you experienced over the last 24hrs?"', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility."}, {'type': 'SECONDARY', 'title': 'Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'OG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At least one month after delivery.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'FG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'FG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'FG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'A total of 26 participants were screened at 25 sites in 3 countries (New Zealand, United Kingdom, and United States). Of them, 4 were enrolled in the study.', 'preAssignmentDetails': 'After a screening period of up to 10 days during which all procedures listed for the screening visit are completed, eligible patients diagnosed with ICP with screening sBA ≥10 μmol/L during screening or at any time during the current pregnancy were randomized with stratification in a 2-arm (1:1), open-label fashion to receive volixibat 20 mg BID or volixibat 80 mg BID. A total of 26 ICP patients were screened for the study. 22 of these patients were screen failures.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '4', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'Part 1 Arm 1 - Volixibat 20mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'BG001', 'title': 'Part 1 Arm 2 - Volixibat 80mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'BG002', 'title': 'Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.\n\nVolixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.'}, {'id': 'BG003', 'title': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.\n\nPlacebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG004'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'White', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG004'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': "Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility."}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-04-20', 'size': 1492048, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-11-30T02:44', 'hasProtocol': True}, {'date': '2020-11-13', 'size': 741712, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-11-30T02:44', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Masking in Part 2 Only'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'whyStopped': "Due to company's assessment of enrollment feasibility.", 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2021-01-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-07', 'completionDateStruct': {'date': '2022-12-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-07-11', 'studyFirstSubmitDate': '2021-01-19', 'resultsFirstSubmitDate': '2023-11-30', 'studyFirstSubmitQcDate': '2021-01-19', 'lastUpdatePostDateStruct': {'date': '2024-08-06', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-07-11', 'studyFirstPostDateStruct': {'date': '2021-01-22', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-08-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assess the Safety and Tolerability of Volixibat in Participants With ICP', 'timeFrame': 'Through to end of treatment, up to 21 weeks', 'description': 'To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints:\n\nProportion of participants experiencing one or more of the following:\n\nTreatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs.\n\nClinically significant laboratory abnormalities'}], 'secondaryOutcomes': [{'measure': 'Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)', 'timeFrame': 'Through to end of treatment, up to 21 weeks', 'description': 'Adult Itch Reported Outcome (ItchRO) is a 0 to 10 scale with 0 being "no itch" and 10 being "worst possible itch" where participants are responding to the following question "How would you rate the worst itch you experienced over the last 24hrs?"'}, {'measure': 'Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes', 'timeFrame': 'At least one month after delivery.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Pruritus', 'Itch', 'Itching', 'Cholestasis', 'Bile Acid', 'Pregnancy', 'Pregnant', 'Intrahepatic', 'Liver', 'ICP'], 'conditions': ['Intrahepatic Cholestasis of Pregnancy']}, 'descriptionModule': {'briefSummary': 'Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat. Part 2 is a double-blind, placebo controlled, study designed to evaluate the safety and efficacy of a selected volixibat dose.'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['ADULT'], 'maximumAge': '45 Years', 'minimumAge': '18 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Female aged ≥18 and ≤45 years with a viable pregnancy.\n2. Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.\n3. Diagnosis of ICP.\n4. (Part 2 only) Qualified level of pruritus associated with ICP, during screening.\n\nExclusion Criteria:\n\n1. At the time of either the screening or baseline visit, decision has already been made to deliver within the next 7 days, for any indication.\n2. Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.\n3. Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.\n4. Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.'}, 'identificationModule': {'nctId': 'NCT04718961', 'acronym': 'OHANA', 'briefTitle': 'A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 2a/2b Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in Adult Women With Intrahepatic Cholestasis of Pregnancy and Elevated Serum Bile Acid Concentrations (OHANA).', 'orgStudyIdInfo': {'id': 'VLX-401'}, 'secondaryIdInfos': [{'id': '2020-003448-96', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part 1 Arm 1 - Volixibat 20mg', 'description': 'Participants randomized to this arm will receive volixibat 20mg twice daily.', 'interventionNames': ['Drug: Volixibat']}, {'type': 'EXPERIMENTAL', 'label': 'Part 1 Arm 2 - Volixibat 80mg', 'description': 'Participants randomized to this arm will receive volixibat 80mg twice daily.', 'interventionNames': ['Drug: Volixibat']}, {'type': 'EXPERIMENTAL', 'label': 'Part 2 Arm 1 - Volixibat Selected Dose mg', 'description': 'Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.', 'interventionNames': ['Drug: Volixibat']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part 2 Arm 2 - Placebo (Placebo Comparator)', 'description': 'Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Volixibat', 'type': 'DRUG', 'otherNames': ['SHP626'], 'description': 'Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.', 'armGroupLabels': ['Part 1 Arm 1 - Volixibat 20mg', 'Part 1 Arm 2 - Volixibat 80mg', 'Part 2 Arm 1 - Volixibat Selected Dose mg']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Capsules matched to study drug minus active substance.', 'armGroupLabels': ['Part 2 Arm 2 - Placebo (Placebo Comparator)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '06511', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale School of Medicine', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Ohio State University Wexner Medical Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '77555', 'city': 'Galveston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Medical Branch - Galveston', 'geoPoint': {'lat': 29.30135, 'lon': -94.7977}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Science Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '78229', 'city': 'San Antonio', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Science Center at San Antonio', 'geoPoint': {'lat': 29.42412, 'lon': -98.49363}}, {'zip': '6021', 'city': 'Dunedin', 'state': 'Otago', 'country': 'New Zealand', 'facility': 'Dunedin Hospital', 'geoPoint': {'lat': -45.87416, 'lon': 170.50361}}, {'zip': '8011', 'city': 'Christchurch', 'country': 'New Zealand', 'facility': "Christchurch Women's Hospital", 'geoPoint': {'lat': -43.53333, 'lon': 172.63333}}, {'zip': '6021', 'city': 'Wellington', 'country': 'New Zealand', 'facility': 'Capital & Coast District Health Board, Wellington Regional Hospital', 'geoPoint': {'lat': -41.28664, 'lon': 174.77557}}, {'zip': 'ME7 5NY', 'city': 'Gillingham', 'state': 'Kent', 'country': 'United Kingdom', 'facility': 'Medway NHS Foundation Trust', 'geoPoint': {'lat': 51.38914, 'lon': 0.54863}}, {'zip': 'BD9 6RJ', 'city': 'Bradford', 'state': 'West Yorkshire', 'country': 'United Kingdom', 'facility': 'Bradford Royal Infirmary', 'geoPoint': {'lat': 53.79391, 'lon': -1.75206}}, {'zip': 'B15 2TG', 'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'Birmingham Womens and Childrens NHS Foundation Trust', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'CF14 4XW', 'city': 'Cardiff', 'country': 'United Kingdom', 'facility': 'University Hospital of Wales', 'geoPoint': {'lat': 51.48, 'lon': -3.18}}, {'zip': 'PO19 6SE', 'city': 'Chichester', 'country': 'United Kingdom', 'facility': "St Richard's Hospital", 'geoPoint': {'lat': 50.83673, 'lon': -0.78003}}, {'zip': 'E11 1NR', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Barts Health NHS Trust- Whipps Cross University Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free London Hospital NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE 1 7EH', 'city': 'London', 'country': 'United Kingdom', 'facility': "Guy's and St Thomas' NHS Foundation Trust", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'TW7 6AF', 'city': 'Middlesex', 'country': 'United Kingdom', 'facility': 'West Middlesex University Hospital', 'geoPoint': {'lat': 51.53174, 'lon': -0.26856}}, {'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'The Newcastle upon Tyne Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}