clinical trial · NCT07223944
A Gaucher Disease Gene Therapy Trial With FLT201
Gaucher Disease Type 1FLT201
brief 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.
started
Apr 7, 2026
primary completion
Jul 1, 2028
completion
Oct 1, 2032
last updated
Apr 21, 2026
official title
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol