A Gaucher Disease Gene Therapy Trial With FLT201
Start Date
4/7/2026
Completion Date
10/1/2032
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.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
FLT201
Conditions
Locations
Kaiser Permanente - CA
Los Angeles, California 90027
United States
UCLA Health
Los Angeles, California 90095
United States
University of California, San Francisco (UCSF)
San Francisco, California 94609
United States
University of Colorado Anschutz
Aurora, Colorado 80045
United States
Emory Healthcare
Atlanta, Georgia 30322
United States
University of Iowa Health Care
Iowa City, Iowa 52242
United States
University of Minnesota
Minneapolis, Minnesota 55454
United States
Hackensack University Medical Center
Hackensack, New Jersey 07601
United States
NYU Langone Health
New York, New York 10017
United States
Columbia University
New York, New York 10032
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
United States
University of Texas Southwestern
Dallas, Texas 75390
United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia 22030-6066
United States
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
Argentina
Hospital Militar Central
Buenos Aires, C1426BOR
Argentina
Clinica Universitaria Reina Fabiola
Córdoba, X5004ASL
Argentina
Melbourne Health - Royal Melbourne Hospital
Melbourne N., Victoria 3051
Australia
Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-903
Brazil
MAGIC Clinic Ltd
Calgary, Alberta T2E 7H7
Canada
Lady Davis Institute for Medical Research Jewish General Hospital
Montreal, Quebec T2E 7Z4
Canada
Shaare Zedek Medical Center
Jerusalem,
Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva,
Israel
King Faisal Specialist Hospital and Research Center
Riyadh, 12713
Saudi Arabia
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona 08035
Spain
Hospital Quironsalud de Zaragoza
Zaragoza, 50012
Spain
Gazi University Medical Faculty
Ankara, Yenimahalle 06560
Turkey (Türkiye)
Queen Elizabeth Hospital Birmingham
Birmingham, Birmingham B15 2GW
United Kingdom
Salford Royal Hospital
Salford, Greater Manchester M6 8HD
United Kingdom
Royal Free Hospital
London, London NW3 2QG
United Kingdom
Addenbrooke's Hospital
Cambridge, CB2 0QQ
United Kingdom