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The goal of this multi-center Phase II study is to assess whether long-term use of rAvPAL-PEG is safe and can maintain reduced blood phenylalanine (Phe) concentrations in patients with Phenylketonuria (PKU). PKU is a genetic disorder characterized by an inability of the body to utilize Phe, an essential amino acid that cannot be produced by the body, but is obtained through food. Phe accumulates in individuals with PKU, causing health problems.
This study is only open to participants who completed participation in an earlier phase of this study (PAL-002).
Males: Ages 16 to 55
Females: Ages 16 to 55
Participants will need to go to the UPMC Montefiore Clinical Translational Research Center at least every week for study drug injections.
Visits: Not applicable
Duration: To be determined
BioMarin Pharmaceutical Inc.
Study description at National Institutes of Health
Phenylketonuria (PKU) Program
Inborn Errors of Metabolism Clinic
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Jessica Lindenberger, 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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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