Progressive Familial Intrahepatic Cholestasis Treatment with Maralixibat – Phase III

MRX-502: Randomized Double-blind Placebo-controlled Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis

Protocol Description

This multi-center research program evaluates the effectiveness, potential side effects and how the body processes the drug maralixibat versus a placebo, when used as a therapy for progressive familial intrahepatic cholestasis (PFIC) in children, adolescents and teens. PFIC is a rare genetic condition in which cells in the liver cannot release bile, a fluid that helps the body digest food. As a result, bile builds up in the liver, leading to symptoms such as jaundice, itching, impaired growth, and delayed puberty.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to patients who have been diagnosed with PFIC.
Males and Females: 1 to 17 years of age


Participants will be randomly assigned to receive the study drug or a placebo as an oral solution to be taken twice a day. Participants or their caregivers will be required each day to complete a dosing diary to confirm when the participant has taken the study drug and will use an electronic device provided by the study team to complete certain questionnaires. Participants will also receive examinations, including blood and urine sampling, and ultrasound imaging at specific intervals.
Visits: 10
Duration: 8 months

Status: Open to Enrollment

Source(s) of Support

Mirum Pharmaceuticals

Contact Information

For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator Amanda Blasko, RN, BSN