A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
NCT ID: NCT04530994
Last Updated: 2021-10-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
APPROVED_FOR_MARKETING
EXPANDED_ACCESS
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
maralixibat
Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Clinical and/or genetic diagnosis of ALGS
3. Male or female participants aged \>12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
4. 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
5. No other therapeutic options or access to any ongoing ALGS clinical trials
Exclusion Criteria
2. Female who is pregnant or breastfeeding
3. 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
4. Past medical history of compensated or decompensated cirrhosis
5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
12 Months
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Clinigen, Inc.
INDUSTRY
Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of California, Los Angeles
Los Angeles, California, United States
Stanford University
Redwood City, California, United States
University of California San Francisco
San Francisco, California, United States
Georgetown University
Washington D.C., District of Columbia, United States
Advent Health
Orlando, Florida, United States
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, United States
University of Minnesota
Minneapolis, Minnesota, United States
The Children's Hospital at Montefiore
New York, New York, United States
University of Oklahoma
Oklahoma City, Oklahoma, United States
Baylor College of Medicine
Houston, Texas, United States
University of Virginia
Charlottesville, Virginia, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Countries
Review the countries where the study has at least one active or historical site.
Related Links
Access external resources that provide additional context or updates about the study.
Genetics Home Reference - Alagille syndrome
US FDA Resources
Mirum Pharmaceuticals homepage
Mirum webpage specific to ALGS EAP
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
MRX-EAP
Identifier Type: -
Identifier Source: org_study_id