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This multi-center research program evaluates the effectiveness, potential side effects, and how the body processes the drug maralixibat when used as a therapy for progressive familial intrahepatic cholestasis (PFIC) in children, adolescents and teens over an extended period of time. 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.
This study (MRX-503) is open to patients who have PFIC and who participated in an earlier maralixibat study protocol (MRX-502).
Males and Females: 1 to 18 years of age
The study drug will be given as an oral solution taken twice a day. Each day, participants or their caregivers will be required to complete study questionnaires using an electronic data logging device provided by the study team. Participants will receive examinations, including blood and urine sampling, and ultrasound imaging at specific intervals.
Duration: 2 years
Study Description at the National Institutes of Health
Progressive Familial Intrahepatic Cholestasis
Robert Squires, MD
For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator Amanda Blasko, RN, BSN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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