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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 and before dinner. 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.
Visits: Study visits every 12 weeks.
Duration: Participants are currently in the optional follow-up treatment period, where they will continue to receive study drug until they enter another LUM001 study or until LUM001 is available by prescription, whichever occurs first.
Study Description at National Institutes of Health
Robert H. Squires, MD
For more information about the study or enrollment, please contact:
Lori Haberstroh, 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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