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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Gene Therapy Study in Patients With Gaucher Disease Type 1


NCTID NCT05324943 (View at clinicaltrials.gov)
Description
Indication Gaucher Disease, Type 1
Compound Name FLT201 (AAVS3-LSP-GBA1-85)
Sponsor Spur Therapeutics
Funder Type Industry
Status
Active not recruiting
Enrollment Count 18

Therapy Information


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

Study Record Dates


Current Stage Phase1
Submit Date 2022-02-28
Completion Date 2025-01-31
Last Update 2024-08-07

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 10
Locations United States,Brazil,United Kingdom,Israel,Germany,Spain

Regulatory Information


Has US IND True
Recent Updates Phase 3 trial planned in 2H2025

Resources/Links