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This multi-center international study will investigate whether the drug maralixibat can improve liver health and delay or reduce the need for a future liver transplant for babies who recently had a hepatoportoenterostomy, also called the Kasai procedure, for biliary atresia. In this rare disease, the ducts that drain bile from the liver are blocked, resulting in a build-up of bile that can cause liver damage. The Kasai procedure bypasses blocked ducts helping the liver to work better, but only delaying the need for a liver transplant. Maralixibat reduces the level of bile in the body, which may help reduce liver damage caused by too much bile.
This study is open to babies diagnosed with biliary atresia who recently had an operation called the Kasai procedure.
Boys and Girls: Babies ages 21 to 111 days
Half the study participants will receive maralixibat daily and the other half will receive a placebo for 6 months. After that, all study participants will receive maralixibat for the remainder of the study. After 2 years, children may be able to continue in the program, unless maralixibat becomes available by prescription, the study is stopped, or the child’s doctor recommends discontinuing participation.
Visits: About 22, many of which will be phone calls rather than in-person visits if specific lab work or other physical examinations are not required
Duration: About 2 years
Study Description at the National Institutes of Health
Simon Horslen, MD
For more information about the study or enrollment, please contact:
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
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