A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
1 other identifier
expanded_access
N/A
1 country
14
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
14 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 25, 2020
CompletedFirst Posted
Study publicly available on registry
August 28, 2020
CompletedOctober 20, 2021
October 1, 2021
August 25, 2020
October 12, 2021
Conditions
Keywords
Interventions
Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor
Eligibility Criteria
You may qualify if:
- Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
- Clinical and/or genetic diagnosis of ALGS
- Male or female participants aged \>12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
- 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
- No other therapeutic options or access to any ongoing ALGS clinical trials
You may not qualify if:
- Diagnosis with a cholestatic liver disease other than ALGS
- Female who is pregnant or breastfeeding
- 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
- Past medical history of compensated or decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Mirum Pharmaceuticals, Inc.lead
- Clinigen, Inc.collaborator
Study Sites (14)
University of California, Los Angeles
Los Angeles, California, 90095, United States
Stanford University
Redwood City, California, 94063, United States
University of California San Francisco
San Francisco, California, 94158, United States
Georgetown University
Washington D.C., District of Columbia, 20007, United States
Advent Health
Orlando, Florida, 32804, United States
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30329, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
The Children's Hospital at Montefiore
New York, New York, 10467, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
University of Virginia
Charlottesville, Virginia, 22903, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 25, 2020
First Posted
August 28, 2020
Last Updated
October 20, 2021
Record last verified: 2021-10