A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
- Conditions
- Alagille Syndrome
- Registration Number
- NCT04530994
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- 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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
- 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
- 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
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (14)
Georgetown University
🇺🇸Washington, District of Columbia, United States
Ochsner Clinic Foundation
🇺🇸New Orleans, Louisiana, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
University of California, Los Angeles
🇺🇸Los Angeles, California, United States
Ann & Robert H. Lurie Children's Hospital
🇺🇸Chicago, Illinois, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
The Children's Hospital at Montefiore
🇺🇸New York, New York, United States
University of Oklahoma
🇺🇸Oklahoma City, Oklahoma, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Advent Health
🇺🇸Orlando, Florida, United States
Children's Healthcare of Atlanta/Emory University
🇺🇸Atlanta, Georgia, United States
Stanford University
🇺🇸Redwood City, California, United States
University of California San Francisco
🇺🇸San Francisco, California, United States