U.S., Nov. 4 -- ClinicalTrials.gov registry received information related to the study (NCT07223944) titled 'A Gaucher Disease Gene Therapy Trial With FLT201' on Oct. 30.
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.
Study Start Date: July 01, 2026
Study Type: INTERVENTIONAL
Condition:
Gaucher Disease Type 1
Intervention:
GENETIC: FLT201
FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-as...