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Trial record 1 of 1 for:    expanded access maralixibat
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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
Clinigen, Inc.
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Alagille Syndrome Drug: maralixibat

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.

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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

Intervention Details:
  • Drug: maralixibat
    Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
  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:

  1. Diagnosis with a cholestatic liver disease other than ALGS
  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

Information from the National Library of Medicine

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

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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
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Clinigen, Inc.
Additional Information:
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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
Keywords provided by Mirum Pharmaceuticals, Inc.:
Liver Disease
Bile Duct Diseases
Digestive System Diseases
Biliary Tract Diseases
Additional relevant MeSH terms:
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Alagille Syndrome
Pathologic Processes
Skin Diseases
Skin Manifestations
Cholestasis, Intrahepatic
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