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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.
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.
Duration: 8 months
Study Description at the National Institutes of Health
Progressive Familial Intrahepatic Cholestasis
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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