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A Gaucher Disease Gene Therapy Trial With FLT201
Sponsor: Spur Therapeutics
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.
Official title: A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
45
Start Date
2026-04-07
Completion Date
2032-10-01
Last Updated
2026-07-13
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (33)
Kaiser Permanente - CA
Los Angeles, California, United States
UCLA Health
Los Angeles, California, United States
University of California, San Francisco (UCSF)
San Francisco, California, United States
University of Colorado Anschutz
Aurora, Colorado, United States
Emory Healthcare
Atlanta, Georgia, United States
University of Iowa Health Care
Iowa City, Iowa, United States
University of Minnesota
Minneapolis, Minnesota, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
NYU Langone Health
New York, New York, United States
Columbia University
New York, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Texas Southwestern
Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
Hospital Militar Central
Buenos Aires, Argentina
Clinica Universitaria Reina Fabiola
Córdoba, Argentina
Melbourne Health - Royal Melbourne Hospital
Melbourne N., Victoria, Australia
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
MAGIC Clinic Ltd
Calgary, Alberta, Canada
Lady Davis Institute for Medical Research Jewish General Hospital
Montreal, Quebec, Canada
Shaare Zedek Medical Center
Jerusalem, Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva, Israel
King Faisal Specialist Hospital and Research Center
Riyadh, Saudi Arabia
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona, Spain
Hospital Quironsalud de Zaragoza
Zaragoza, Spain
Gazi University Medical Faculty
Ankara, Yenimahalle, Turkey (Türkiye)
Queen Elizabeth Hospital Birmingham
Birmingham, Birmingham, United Kingdom
Salford Royal Hospital
Salford, Greater Manchester, United Kingdom
Royal Free Hospital
London, London, United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom