Children's Hospital is part of the UPMC family.
This multi-center trial studies the effectiveness and long-term safety and tolerability of the drug LUM001 when taken by children with Progressive Familial Intrahepatic Cholestasis (PFIC). Researchers will study the drug’s effect on pruritis (itch), serum bile acids, and biochemical markers in pediatric patients with PFIC.
Subject to certain exclusion criteria, such as biliary diversion or prior liver transplant, the study is open to males and females, ages 12 months through 18 years with a confirmed diagnosis of PFIC.
Males and Females: 12 months through 18 years
Following an initial screening visit in which the child is given a comprehensive physical, including blood work, patients will be provided an electronic diary in which they or their parents will record the intensity of itching in the morning and before they go to bed. Study participants will be provided with the study drug to be taken each day before breakfast. At various intervals a research coordinator will contact parents and/or participants to get updates and see how the child is doing on the study drug. Return visits will be scheduled for physicals, blood and urine sampling, at specific intervals. There is also an optional 52-week extension to the study for patients that wish to continue past the 76 weeks.
Visits: Approximately 12
Duration: 76 weeks
Study Description at National Institutes of Health
Robert H. Squires, MD
For more information about the study or enrollment, please contact:
Lori Haberstroh, RN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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