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].



Eligible Ages
Between 0 Days and 364 Days
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

  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

Exclusion Criteria

  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

Study Design

Phase 2
Study Type
Intervention Model
Single Group Assignment
Intervention Model Description
Single group with 2 cohorts - ≥ 6 participants each from ALGS and PFIC.
Primary Purpose
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
  • Drug: Maralixibat
    Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base
    Other names:
    • Formerly LUM001 and SHP625

Recruiting Locations

Medstar Georgetown University Hospital
Washington, District of Columbia 20007
Carissa Vinovskis

More Details

Mirum Pharmaceuticals, Inc.

Study Contact

Clinical Trials Mirum

Detailed Description

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


Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.