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

NCTID	NCT04046224 (View at clinicaltrials.gov)
Description	This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the 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. (Show More)
Development Status	Active
Indication	Fabry Disease
Disease Ontology Term	DOID:14499
Compound Name	ST-920
Compound Alias	Isaralgagene civaparvovec
Sponsor	Sangamo Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	34
Results Posted	Not Available

Target Gene/Variant	GLA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/6
Editor Type	none
Dose 1	0.26E13 vg/kg
Dose 2	0.53E13 vg/kg
Dose 3	1.58E13 vg/kg
Dose 4	2.63E13 vg/kg (expansion dose)
Dose 5

No.of Trial Sites	18
Locations	Canada,United States,Taiwan,Italy,United Kingdom,Australia,Germany

Has US IND	True
FDA Designations	Accelerated Approval, Orphan Drug Designation
Recent Updates	Recent Type B meeting with FDA allowed ongoing Phase 1/2 study to serve as the primary basis for approval under Accelerated Approval Program, BLA submission H2 2025