NCT04046224

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease (STAAR)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Sangamo Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	ST-920	Single dose of investigational product ST-920

Timeline

• Start date: 2019-07-23
• Primary completion: 2025-04-10
• Completion: 2025-04-10
• First posted: 2019-08-06
• Last updated: 2026-04-14
• Results posted: 2026-04-14

Locations

18 sites across 7 countries: United States, Australia, Canada, Germany, Italy, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study