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In this study, researchers are evaluating a drug called DCR-PHXC, when given to children and adults with primary hyperoxaluria (PH) type 1 or type 2 (PH1 or PH2). More specifically, researchers will be studying how well the drug helps the liver to make less oxalate to reduce symptoms of PH. Lowering the levels of oxalate may have a positive effect on patients with PH because most of the symptoms from PH are due to the excessive amounts of oxalate accumulating in the body.
Subject to certain exclusion criteria, this study is accepting participants, ages 6 and older who have been diagnosed with PH1 or PH2.
Males and Females: Ages 6 and older
Participants will be randomly assigned to be given either the study drug or a placebo. The drug will be administered monthly by injection during study visits. There will be a total of 12 study visits for examinations, medical tests, and imaging.
Duration: Up to 7 months
Study Description at National Institutes of Health
Michael Moritz, MD
For more information about the study or enrollment, please contact:
Vibha Chauhan, PhD, CCRP, CCRC
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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