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). (RISE)

Mirum Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Progressive Familial Intrahepatic Cholestasis; Cholestatic Liver Disease; Alagille Syndrome

Treatments: Drug: Maralixibat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04729751

2020-004628-40 (EudraCT Number)

MRX-801

Details and patient eligibility

About

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].

Full description

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

Enrollment: 27 patients

Sex: All

Ages: Under 364 days old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 1 patient group

Maralixibat

Experimental group

Description:

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.

Treatment:

Drug: Maralixibat