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This multi-center international study is being done to see how the study drug, PTC923, affects blood phenylalanine (Phe) levels in the body of people with phenylketonuria (PKU), a rare inherited disorder that causes the amino acid Phe to build up in the body when protein is consumed. Researchers believe that PTC923 may help to reduce the amount of Phe in the body.
Subject to certain exclusion criteria, this study is accepting people with PKU of all ages, starting with newborns.
Males and Females: All ages
Following an initial screening period, which will include physical examinations and blood testing, participants will begin Part 1 of the study program. During Part 1, all participants will take PTC923 for 2 weeks, with blood Phe levels monitored to see if there is a response to the study drug. Only those participants who respond to PTC923 treatment will remain in the study for Part 2. In this 6-week phase, participants will either be assigned to take a placebo or the study drug at a dosage level that will be increased every 2 weeks.
Duration: About 4 months
PTC Therapeutics Inc.
Study Description at National Institutes of Health
Genetic and Genomic Medicine Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
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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