Trials / Completed
CompletedNCT05324943
A Gene Therapy Study in Patients With Gaucher Disease Type 1
A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- Spur Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.
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
- 2022-04-15
- Primary completion
- 2024-12-04
- Completion
- 2024-12-04
- First posted
- 2022-04-13
- Last updated
- 2025-11-04
Locations
10 sites across 6 countries: United States, Brazil, Germany, Israel, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
- FDA-regulated device study
Source: ClinicalTrials.gov record NCT05324943. Inclusion in this directory is not an endorsement.