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This multi-center international study is being done to investigate a potential alternative treatment for Fabry disease using a single-dose of the drug ST-920, a form of gene therapy. The goal of gene therapy is to deliver a healthy gene that will help the body to produce an enzyme that was previously deficient due to Fabry disease, a rare condition caused by mutations in the GLA gene, which normally produces the protein alpha-galactosidase A (α-Gal A). When α-Gal A is missing or not working properly, fat and related substances accumulate in organs, vessels, nerves, and tissues throughout the body, leading to diseases that impact blood vessels, brain, kidneys, heart, and skin, among others. The current standards of care are life-long enzyme replacement therapy and pharmacological chaperone therapy that improves function of the enzyme produced by the body.
Subject to certain exclusion criteria, this study is accepting people with Fabry disease, ages 18 and older. Males and Females: Ages 18 years and older
After an 8-week screening process and 12 weeks of baseline monitoring, including physicals and other testing, study participants will receive a single dose of ST-920 at one of 4 dosing levels at UPMC Children’s Hospital of Pittsburgh. After discharge from the hospital, participants will be followed for about a year, with scheduled physicals, health monitoring, and additional testing to measure effects and side effects of the study drug. Based on outcomes of the initial enrollees, researchers will determine when to proceed to higher dosing levels for other participants. Visits: About 17 Duration: About 17 months
Sangamo Therapeutics, Inc.
Study Description at National Institutes of Health Fabry Disease Genetic and Genomic Medicine Research
Damara Ortiz, MD
For more information about the study or enrollment, please contact: Nadene Henderson, MS, LCGC 412-692-3475
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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