for people ages 12 months and up (full criteria)
at San Francisco, California and other locations
study started



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 or telephone +1-877-768-4303.

Official Title

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome


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.


Alagille Syndrome, Liver Disease, ALGS, Maralixibat, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritis, Pruritus, Syndrome


You can join if…

Open to people ages 12 months and up

  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

You CAN'T join if...

  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


  • UCSF
    San Francisco California 94158 United States
  • Stanford University
    Redwood City California 94063 United States


not accepting new patients
Start Date
Mirum Pharmaceuticals, Inc.
Genetics Home Reference - Alagille syndrome Mirum Pharmaceuticals homepage
Study Type
Expanded Access
Last Updated