Clinical trialCLINICALTRIALSApr 14
Trial Results Posted: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) (NCT04046224)
Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.
WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.