Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Ranked #6 Nationally by U.S. News & World Report.
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
Genetic and Genomic Medicine Research
Damara Ortiz, MD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.