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This multi-center research study evaluates the effectiveness, potential side effects, and how the body processes the drug maralixibat when used to treat patients with cholestatic liver disease over an extended period of time.
Subject to certain exclusion criteria, this study is open to patients with cholestatic liver disease who previously participated in and completed a maralixibat study.
Males and Females: 12 months and older
The study drug will be given as an oral solution taken once or twice a day as determined by the study team. Each day, participants or their caregivers will be required to complete study questionnaires via their smartphone or tablet. Participants will return to clinic every 16 weeks for examinations, including blood and urine sampling, and imaging. Participants may remain in the study until the drug is commercially available, the study ends, or the patient is withdrawn from the study for other reasons.
Visits: Every 4 months
Duration: Open ended
Study Description at the National Institutes of Health
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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