A Gaucher Disease Gene Therapy Trial With FLT201

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.

Timeline

Start

2026-04-07

Primary completion

2028-07-01

Completion

2032-10-01

Drugs

Indications