A Gaucher Disease Gene Therapy Trial With FLT201
Recruiting now · Phase 3
Conditions studied: Gaucher Disease Type 1
In brief
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.
Key facts
- Study ID
- NCT07223944
- Run by
- Spur Therapeutics
- People needed
- 45
- Starts
- 2026-04-07
- Expected to finish
- 2032-10-01
- Last updated by the study team
- 2026-07-13
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Kaiser Permanente - CA — Los Angeles, California, United States (enrolling)
- University of California, San Francisco (UCSF) — San Francisco, California, United States (enrolling)
- University of Colorado Anschutz — Aurora, Colorado, United States (enrolling)
- University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
- Duke University Medical Center — Durham, North Carolina, United States (enrolling)
- University of Texas Southwestern — Dallas, Texas, United States (enrolling)
- Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States (enrolling)
- Hospital Italiano de Buenos Aires — Buenos Aires, Argentina (enrolling)
- Clinica Universitaria Reina Fabiola — Córdoba, Argentina (enrolling)
- MAGIC Clinic Ltd — Calgary, Alberta, Canada (enrolling)
- Shaare Zedek Medical Center — Jerusalem, Israel (enrolling)
- Rabin Medical Center, Beilinson Hospital — Petah Tikva, Israel (enrolling)
- Royal Free Hospital — London, London, United Kingdom (enrolling)
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- King Faisal Specialist Hospital and Research Center — Riyadh, Saudi Arabia
- Hospital Universitario Vall d'Hebron — Barcelona, Barcelona, Spain
- Hospital Quironsalud de Zaragoza — Zaragoza, Spain
- Hospital Militar Central — Buenos Aires, Argentina
- Gazi University Medical Faculty — Ankara, Yenimahalle, Turkey (Türkiye)
- Melbourne Health - Royal Melbourne Hospital — Melbourne N., Victoria, Australia
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Brazil
- UCLA Health — Los Angeles, California, United States
- Queen Elizabeth Hospital Birmingham — Birmingham, Birmingham, United Kingdom
- Lady Davis Institute for Medical Research Jewish General Hospital — Montreal, Quebec, Canada
- Emory Healthcare — Atlanta, Georgia, United States
Full record on ClinicalTrials.gov
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