Ignite Creation Date:
2025-12-25 @ 1:27 AM
Ignite Modification Date:
2025-12-27 @ 11:25 PM
Study NCT ID:
NCT04530994
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2021-10-20
First Post:
2020-08-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016738', 'term': 'Alagille Syndrome'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D002779', 'term': 'Cholestasis'}, {'id': 'D011537', 'term': 'Pruritus'}], 'ancestors': [{'id': 'D002780', 'term': 'Cholestasis, Intrahepatic'}, {'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'}, {'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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000722912', 'term': 'maralixibat'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'treatment': True}, 'nPtrsToThisExpAccNctId': 1}, 'statusModule': {'overallStatus': 'APPROVED_FOR_MARKETING', 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-10', 'lastUpdateSubmitDate': '2021-10-12', 'studyFirstSubmitDate': '2020-08-25', 'studyFirstSubmitQcDate': '2020-08-25', 'lastUpdatePostDateStruct': {'date': '2021-10-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-08-28', 'type': 'ACTUAL'}}, 'conditionsModule': {'keywords': ['Liver Disease', 'ALGS', 'Maralixibat', 'Bile Duct Diseases', 'Digestive System Diseases', 'Biliary Tract Diseases', 'Cholestasis', 'Pruritis'], 'conditions': ['Alagille Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://ghr.nlm.nih.gov/condition/alagille-syndrome', 'label': 'Genetics Home Reference - Alagille syndrome'}, {'url': 'https://clinicaltrials.gov/ct2/info/fdalinks', 'label': 'US FDA Resources'}, {'url': 'https://mirumpharma.com', 'label': 'Mirum Pharmaceuticals homepage'}, {'url': 'https://algseap.com/', 'label': 'Mirum webpage specific to ALGS EAP'}]}, 'descriptionModule': {'briefSummary': 'An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.', 'detailedDescription': 'The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Months', 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)\n2. Clinical and/or genetic diagnosis of ALGS\n3. Male or female participants aged \\>12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS\n4. Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program\n5. No other therapeutic options or access to any ongoing ALGS clinical trials\n\nExclusion Criteria:\n\n1. Diagnosis with a cholestatic liver disease other than ALGS\n2. Female who is pregnant or breastfeeding\n3. Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program\n4. Past medical history of compensated or decompensated cirrhosis\n5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action'}, 'identificationModule': {'nctId': 'NCT04530994', 'briefTitle': 'A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mirum Pharmaceuticals, Inc.'}, 'officialTitle': 'A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome', 'orgStudyIdInfo': {'id': 'MRX-EAP'}}, 'armsInterventionsModule': {'interventions': [{'name': 'maralixibat', 'type': 'DRUG', 'description': 'Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor'}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94063', 'city': 'Redwood City', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.48522, 'lon': -122.23635}}, {'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': 'Georgetown University', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '32804', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Advent Health', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '30329', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare of Atlanta/Emory University", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '70121', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Ochsner Clinic Foundation', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '10467', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': "The Children's Hospital at Montefiore", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '73104', 'city': 'Oklahoma City', 'state': 'Oklahoma', 'country': 'United States', 'facility': 'University of Oklahoma', 'geoPoint': {'lat': 35.46756, 'lon': -97.51643}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor College of Medicine', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '22903', 'city': 'Charlottesville', 'state': 'Virginia', 'country': 'United States', 'facility': 'University of Virginia', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Medical College of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mirum Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Clinigen, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}