at San Francisco, California and other locations
study started
estimated completion
Principal Investigator
by Philip Rosenthal



This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Official Title

Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome


This is an open label study where all participants will receive maralixibat treatment.


Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease, PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Pediatric, Cholestasis, Intrahepatic Cholestasis, Syndrome


You can join if…

  1. Body weight of ≥2.5 kg
  2. <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
  3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
  4. Diagnosis of PFIC or ALGS

You CAN'T join if...

  1. Predicted complete absence of bile salt excretion pump (BSEP) function
  2. History of surgical disruption of the enterohepatic circulation
  3. History of liver transplant or imminent need for liver transplant
  4. Decompensated cirrhosis
  5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
  6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study


  • University of California - San Francisco accepting new patients
    San Francisco California 94158 United States
  • Children Hospital LA accepting new patients
    Los Angeles California 90027 United States

Lead Scientist at UCSF


accepting new patients
Start Date
Completion Date
Mirum Pharmaceuticals, Inc.
Genetics Home Reference - PFIC Mirum Pharmaceuticals homepage Genetics Home Reference - ALGS
Phase 2 research study
Study Type
Expecting 12 study participants
Last Updated