Trials / Not Yet Recruiting
Not Yet RecruitingNCT07223944
A Gaucher Disease Gene Therapy Trial With FLT201
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
- Status
- Not Yet Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 45 (estimated)
- Sponsor
- Spur Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | FLT201 | FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid. |
Timeline
- Start date
- 2026-07-01
- Primary completion
- 2028-07-01
- Completion
- 2032-10-01
- First posted
- 2025-11-03
- Last updated
- 2025-11-03
Regulatory
- FDA-regulated drug study
- FDA-regulated device study
Source: ClinicalTrials.gov record NCT07223944. Inclusion in this directory is not an endorsement.