A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

Purpose

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].

Conditions

  • Progressive Familial Intrahepatic Cholestasis
  • Alagille Syndrome
  • Cholestatic Liver Disease

Eligibility

Eligible Ages
Between 0 Days and 364 Days
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Maralixibat 		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

More Details

Status
Active, not recruiting
Sponsor
Mirum Pharmaceuticals, Inc.

Study Contact

Detailed Description

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