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)
WHY IT MATTERS
This 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.
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.
NCT ID: NCT04046224 Status: COMPLETED Conditions: Fabry Disease Phase: PHASE1, PHASE2 Enrollment: 36 Sponsor: Sangamo Therapeutics Summary: This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry
ASK YOUR DOCTOR
Ask your Fabry disease specialist if you're eligible for ST-920 trials or if results from STAAR might affect your current treatment plan.