Official Title
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
Brief Summary

An open-label, single-arm, multi-centre program providing treatment access to ALGSpatients with cholestatic pruritus in the US. Access can be requested through ClinigenHealthcare 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.

Approved for marketing
Treatment IND/Protocol
Alagille Syndrome

Drug: maralixibat

Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

Eligibility Criteria

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

Eligibility Gender
All
Eligibility Age
Minimum: 12 Months ~ Maximum: N/A
Countries
United States
Locations

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, 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

Clinigen, Inc.
NCT Number
Keywords
Liver Disease
ALGS
Maralixibat
Bile Duct Diseases
Digestive System Diseases
Biliary Tract Diseases
Cholestasis
Pruritis
MeSH Terms
Alagille Syndrome
Pruritus
Syndrome