Status: Recruiting
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.
Participants needed: 45
Trial details
Phase: Phase 3Age: 18+Biological sex: AllType: InterventionalSponsor: Spur TherapeuticsUpdated: Jul 13, 2026Locations: 33
Eligibility criteria
Aged ≥18 years at time of screening. [+4]
Diagnosed or suspected Gaucher disease type 2 or type 3 [+6]