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A Gaucher Disease Gene Therapy Trial With FLT201
Sponsor: Spur Therapeutics
Summary
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.
Official title: A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
45
Start Date
2026-04-07
Completion Date
2032-10-01
Last Updated
2026-04-21
Healthy Volunteers
No
Conditions
Interventions
FLT201
FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
Locations (3)
Duke University Medical Center
Durham, North Carolina, United States
University of Texas Southwestern
Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States