Fabry Disease Treatment with ST-920 Gene Therapy – Phase I/II

Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease

Protocol Description

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.

Study Level: Phase I/II

Eligibility Criteria

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

Status: Open to Enrollment

Source(s) of Support

Sangamo Therapeutics, Inc.

Primary Investigator

Damara Ortiz, MD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC