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The objective of this international multi-center study is to see if the drug pegzilarginase can help people with arginase 1 deficiency. Arginase 1 deficiency is a rare disease caused by a defect in a specific gene that causes a deficiency of the enzyme called arginase 1 in the body. This enzyme’s job is to help break down the amino acid arginine. When arginase is not working, arginine can build up in the blood and can lead to serious effects on growth, health, and intellect.
Subject to certain exclusion criteria, candidates must have a confirmed diagnosis of arginase 1 deficiency.
Males and Females: Ages 2 years and older
Participants will be randomly assigned to a group that receives either a placebo or the study drug via intravenous infusion, weekly for 24 weeks. After that period all participants will receive weekly infusions of pegzilarginase for an additional 150 weeks along with individualized disease management.
Visits: About 178
Duration: About 3-1/2 years
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Jessica Lindenberger, MSN, CRNP, FNP-C
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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