ClinicaliQ Trial Snapshot
- A Gaucher Disease Gene Therapy Trial With FLT201 — Recruiting • Phase III • Rare Diseases • NCT07223944.
- Gene therapy FLT201 tested in Gaucher disease patients already on standard treatment, assessing whether it allows safe discontinuation of current medications.
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What This Trial Is Studying
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. Conditions: Gaucher Disease Type 1 Interventions: FLT201 Lead Sponsor: Spur Therapeutics Planned Enrollment: 45 participants