MedPath

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
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

Study & Design

Study Type
EXPANDED_ACCESS
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (14)

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

Scroll for more (4 remaining)
University of California, Los Angeles
🇺🇸Los Angeles, California, United States

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.