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For more information about research, please call our main office at 412-692-6438.
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This multi-center international study will test maralixibat in infants with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis (PFIC). Maralixibat has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of pruritus (itch) associated with ALGS in patients 1 year of age and older. This research studies the safety and tolerability of maralixibat, as well as its effects and side effects in children less than 12 months of age.
Subject to certain exclusion criteria, this study is open to infants diagnosed with ALGS or PFIC.
Boys and Girls: Newborns and babies less than 1 year old
For potential participants, the initial 4 weeks of the study will determine eligibility. Those accepted will receive the study drug once daily in a liquid oral form and attend regular clinic visits for 13 weeks. After that, participants may continue in the program for a longer-term extension period.
Visits: About 10 for the core study; Every 16 weeks for participants who remain in the long-term study after turning 1
Duration: 17 weeks to several years
Study Description at the National Institutes of Health
Simon Horslen, MD
For more information about the study or enrollment, please contact:
Laura Mathews, Clinical Research Coordinator
Susan Richey, RN, Clinical Research Nurse Coordinator
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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