Ignite Creation Date:
2025-12-24 @ 11:57 PM
Ignite Modification Date:
2025-12-25 @ 9:53 PM
Study NCT ID:
NCT04729751
Status:
COMPLETED
Last Update Posted:
2025-02-06
First Post:
2021-01-25
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-12-18', 'mcpReleaseN': 18, 'releaseDate': '2025-12-03'}, {'releaseDate': '2025-12-23'}], 'estimatedResultsFirstSubmitDate': '2025-12-03'}}, 'conditionBrowseModule': {'meshes': [{'id': 'C535933', 'term': 'Cholestasis, progressive familial intrahepatic 1'}, {'id': 'D016738', 'term': 'Alagille Syndrome'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}], 'ancestors': [{'id': 'D002780', 'term': 'Cholestasis, Intrahepatic'}, {'id': 'D002779', 'term': 'Cholestasis'}, {'id': 'D004066', 'term': 'Digestive System 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': 'C000722912', 'term': 'maralixibat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Single group with 2 cohorts - ≥ 6 participants each from ALGS and PFIC.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-09-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2024-12-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-02-04', 'studyFirstSubmitDate': '2021-01-25', 'studyFirstSubmitQcDate': '2021-01-27', 'lastUpdatePostDateStruct': {'date': '2025-02-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-01-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-12-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency of treatment-emergent adverse events [TEAEs]', 'timeFrame': 'From Baseline through to Week 13'}], 'secondaryOutcomes': [{'measure': 'Change in fasting serum bile acid (sBA) levels', 'timeFrame': 'From Baseline through to Week 13'}, {'measure': 'To evaluate the effect on liver enzymes (ALT, AST) and bilirubin', 'timeFrame': 'From Baseline through to Week 13'}, {'measure': 'To evaluate the effect on LSVs', 'timeFrame': 'From Baseline through to Week 13'}, {'measure': 'To assess the plasma level of maralixibat in infant participants', 'timeFrame': 'At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PFIC', 'ALGS', 'Maralixibat', 'Bile Duct Diseases', 'Liver Diseases', 'Biliary Tract Diseases', 'Pediatric'], 'conditions': ['Progressive Familial Intrahepatic Cholestasis', 'Alagille Syndrome', 'Cholestatic Liver Disease']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://medlineplus.gov/genetics/condition/progressive-familial-intrahepatic-cholestasis/', 'label': 'Genetics Home Reference - PFIC'}, {'url': 'https://mirumpharma.com', 'label': 'Mirum Pharmaceuticals homepage'}, {'url': 'https://medlineplus.gov/genetics/condition/alagille-syndrome/', 'label': 'Genetics Home Reference - ALGS'}, {'url': 'https://clinicaltrials.gov/ct2/info/fdalinks', 'label': 'US FDA Resources'}]}, 'descriptionModule': {'briefSummary': 'This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children \\<12 months of age with Alagille Syndrome \\[ALGS\\] or Progressive Familial Intrahepatic Cholestasis \\[PFIC\\].', 'detailedDescription': 'This is an open label study where all participants will receive maralixibat treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '364 Days', 'minimumAge': '0 Days', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Body weight of ≥2.5 kg\n2. \\<12 months of age at the baseline visit (ROW). \\>31 days and \\<12 months of age at the baseline visit (US).\n3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.\n4. Diagnosis of PFIC or ALGS\n\nExclusion criteria:\n\n1. Predicted complete absence of bile salt excretion pump (BSEP) function\n2. History of surgical disruption of the enterohepatic circulation\n3. History of liver transplant or imminent need for liver transplant\n4. Decompensated cirrhosis\n5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion\n6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study"}, 'identificationModule': {'nctId': 'NCT04729751', 'acronym': 'RISE', 'briefTitle': 'A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome', 'orgStudyIdInfo': {'id': 'MRX-801'}, 'secondaryIdInfos': [{'id': '2020-004628-40', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Maralixibat', 'description': 'Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.', 'interventionNames': ['Drug: Maralixibat']}], 'interventions': [{'name': 'Maralixibat', 'type': 'DRUG', 'otherNames': ['Formerly LUM001 and SHP625'], 'description': 'Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL)\n\n* 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base\n* 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base', 'armGroupLabels': ['Maralixibat']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Children Hospital LA', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California - San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '20007', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': 'Medstar Georgetown University Hospital', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '70121', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Ochsner Hospital for Children', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'city': 'Brussels', 'country': 'Belgium', 'facility': 'Cliniques Universitaires Saint-Luc', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '01308-000', 'city': 'São Paulo', 'country': 'Brazil', 'facility': 'Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'city': 'Le Kremlin-Bicêtre', 'country': 'France', 'facility': 'Hôpital Kremlin Bicêtre', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'city': 'Paris', 'country': 'France', 'facility': 'Hopital Necker', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '45050', 'city': 'Zapopan', 'country': 'Mexico', 'facility': 'Consultorio de Joshue David Covarrubias Esquer', 'geoPoint': {'lat': 20.72111, 'lon': -103.38742}}, {'city': 'Warsaw', 'country': 'Poland', 'facility': 'Instytut Pomnik-Centrum Zdrowia Dziecka', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'city': 'London', 'country': 'United Kingdom', 'facility': "King's College Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}