A Gaucher Disease Gene Therapy Trial With FLT201

NCTID	NCT07223944 (View at clinicaltrials.gov)
Description	This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT. (Show More)
Development Status	Active
Indication	Gaucher Disease Type 1
Disease Ontology Term	DOID:0110957
Compound Name	FLT201
Compound Alias	avigbagene parvec
Sponsor	Spur Therapeutics
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	45 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	GBA1
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	AAVS3
Editor Type	none
Dose 1	4.5E11 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites
Locations

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Trial initiation expected 2H26