A Gaucher Disease Gene Therapy Trial With FLT201

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.

NCT Number	NCT07223944
Lead Sponsor	Spur Therapeutics
Conditions	Gaucher Disease Type 1
Enrollment	45 participants
Start Date	2026-04-07
Primary Completion	2028-07-01 (estimated)
Study Completion	2032-10-01 (estimated)
Updated on ClinicalTrials.gov	2026-08-14