A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
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ClinicalTrials.gov Identifier: NCT04530994 |
Expanded Access Status :
Approved for marketing
First Posted : August 28, 2020
Last Update Posted : October 20, 2021
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Condition or disease | Intervention/treatment |
---|---|
Alagille Syndrome | Drug: maralixibat |
Study Type : | Expanded Access |
Expanded Access Type : | Treatment IND/Protocol |
See clinical trials of the intervention/treatment in this expanded access record. | |
Official Title: | A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome |
- Drug: maralixibat
Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Months and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04530994
United States, California | |
University of California, Los Angeles | |
Los Angeles, California, United States, 90095 | |
Stanford University | |
Redwood City, California, United States, 94063 | |
University of California San Francisco | |
San Francisco, California, United States, 94158 | |
United States, District of Columbia | |
Georgetown University | |
Washington, District of Columbia, United States, 20007 | |
United States, Florida | |
Advent Health | |
Orlando, Florida, United States, 32804 | |
United States, Georgia | |
Children's Healthcare of Atlanta/Emory University | |
Atlanta, Georgia, United States, 30329 | |
United States, Illinois | |
Ann & Robert H. Lurie Children's Hospital | |
Chicago, Illinois, United States, 60611 | |
United States, Louisiana | |
Ochsner Clinic Foundation | |
New Orleans, Louisiana, United States, 70121 | |
United States, Minnesota | |
University of Minnesota | |
Minneapolis, Minnesota, United States, 55455 | |
United States, New York | |
The Children's Hospital at Montefiore | |
New York, New York, United States, 10467 | |
United States, Oklahoma | |
University of Oklahoma | |
Oklahoma City, Oklahoma, United States, 73104 | |
United States, Texas | |
Baylor College of Medicine | |
Houston, Texas, United States, 77030 | |
United States, Virginia | |
University of Virginia | |
Charlottesville, Virginia, United States, 22903 | |
United States, Wisconsin | |
Medical College of Wisconsin | |
Milwaukee, Wisconsin, United States, 53226 |
Responsible Party: | Mirum Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT04530994 |
Other Study ID Numbers: |
MRX-EAP |
First Posted: | August 28, 2020 Key Record Dates |
Last Update Posted: | October 20, 2021 |
Last Verified: | October 2021 |
Liver Disease ALGS Maralixibat Bile Duct Diseases |
Digestive System Diseases Biliary Tract Diseases Cholestasis Pruritis |
Alagille Syndrome Pruritus Syndrome Disease Pathologic Processes Skin Diseases Skin Manifestations Cholestasis, Intrahepatic Cholestasis Bile Duct Diseases |
Biliary Tract Diseases Digestive System Diseases Liver Diseases Heart Defects, Congenital Cardiovascular Abnormalities Cardiovascular Diseases Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, Inborn |