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Through this trial researchers will study the drug LUM001 and its effects on children with cholestatic liver disease with Alagille Syndrome when taken over an extended period. Investigators believe that LUM001 may help control itching and improve liver tests. As a study extension, this trial is only open to participants of another Alagille Syndrome LUM001 trial, known as the ITCH study. The LUM001 studies are being done as a collaboration of the Childhood Liver Disease Research Network centers.
Subject to certain exclusion criteria, the study is open to individuals who have been diagnosed with Alagille Syndrome and evidence of cholestasis with itching. All participants must have completed participation in the ITCH study.
Males and Females: 12 months through 18 years
All participants will receive a daily oral dose of LUM001, and an assessment of each participant’s itching will be logged via electronic diary. Dose levels will be adjusted for optimum effect in the first part of the approximately 11-month-long study. Medical record information and blood and urine samples will also be collected for each participant.
Duration: Up to 48 weeks
National Institute of Diabetes and Digestive and Kidney Diseases
Study Description at National Institutes of Health
Childhood Liver Disease Research Network
Robert H. Squires, MD
For more information about the study or enrollment, please contact:
Kathy Bukauskas, RN, CCRC
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Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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