At a glance
ClinicalIndex Comparison Record- ✓Age ≥18 years
- ✓Documented diagnosis of Fabry disease
- ✓At least one Fabry-related symptom: cornea verticillata, acroparesthesia, anhidrosis, or angiokeratoma
- ✓[Renal cohort] eGFR 40-90 mL/min/1.73 m² and documented declining renal function (≥2 mL/min/1.73m²/year) on ≥3 creatinine values within 18 months
- ✕Presence of neutralizing antibodies to AAV6
- ✕eGFR <40 mL/min/1.73m²
- ✕NYHA Class III or higher heart failure
- ✕Active infection with hepatitis A, B, C, HIV, or tuberculosis
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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)
In Brief
A Phase 2 clinical trial evaluating ST-920 for Fabry Disease. Completed, enrolled 36 participants across 18 sites in 7 countries.
Detailed 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.
Study Details
Timeline
Interventions
Single dose of investigational product ST-920