A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Status: Recruiting
Location: See all (33) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Aged ≥18 years at time of screening.
• Clinical diagnosis of Gaucher disease type 1
• Stable hemoglobin concentration at baseline
• Stable platelet count at baseline
• Receiving ERT or SRT without interruption for at least 2 years
Locations
United States
California
Kaiser Permanente - CA
NOT_YET_RECRUITING
Los Angeles
UCLA Health
NOT_YET_RECRUITING
Los Angeles
University of California, San Francisco (UCSF)
NOT_YET_RECRUITING
San Francisco
Colorado
University of Colorado Anschutz
NOT_YET_RECRUITING
Aurora
Georgia
Emory Healthcare
NOT_YET_RECRUITING
Atlanta
Iowa
University of Iowa Health Care
NOT_YET_RECRUITING
Iowa City
Minnesota
University of Minnesota
RECRUITING
Minneapolis
North Carolina
Duke University Medical Center
RECRUITING
Durham
New Jersey
Hackensack University Medical Center
NOT_YET_RECRUITING
Hackensack
New York
Columbia University
NOT_YET_RECRUITING
New York
NYU Langone Health
NOT_YET_RECRUITING
New York
Ohio
Cincinnati Children's Hospital Medical Center
NOT_YET_RECRUITING
Cincinnati
Pennsylvania
Children's Hospital of Philadelphia
NOT_YET_RECRUITING
Philadelphia
UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITING
Pittsburgh
Texas
University of Texas Southwestern
RECRUITING
Dallas
Virginia
Lysosomal Rare Disorders Research and Treatment Center
RECRUITING
Fairfax
Other Locations
Argentina
Hospital Italiano de Buenos Aires
NOT_YET_RECRUITING
Buenos Aires
Hospital Militar Central
NOT_YET_RECRUITING
Buenos Aires
Clinica Universitaria Reina Fabiola
NOT_YET_RECRUITING
Córdoba
Australia
Melbourne Health - Royal Melbourne Hospital
NOT_YET_RECRUITING
Melbourne N.
Brazil
Hospital de Clinicas de Porto Alegre
NOT_YET_RECRUITING
Porto Alegre
Canada
MAGIC Clinic Ltd
NOT_YET_RECRUITING
Calgary
Lady Davis Institute for Medical Research Jewish General Hospital
NOT_YET_RECRUITING
Montreal
Israel
Shaare Zedek Medical Center
RECRUITING
Jerusalem
Tel Aviv Sourasky Medical Center
RECRUITING
Tel Aviv
Saudi Arabia
King Faisal Specialist Hospital and Research Center
NOT_YET_RECRUITING
Riyadh
Spain
Hospital Universitario Vall d'Hebron
NOT_YET_RECRUITING
Barcelona
Hospital Quironsalud de Zaragoza
NOT_YET_RECRUITING
Zaragoza
Turkey
Gazi University Medical Faculty
NOT_YET_RECRUITING
Ankara
United Kingdom
Queen Elizabeth Hospital Birmingham
NOT_YET_RECRUITING
Birmingham
Addenbrooke's Hospital
NOT_YET_RECRUITING
Cambridge
Royal Free Hospotal
NOT_YET_RECRUITING
London
Salford Royal Hospital
NOT_YET_RECRUITING
Salford
Contact Information
Primary
Spur Clinical Trials Contact
clinicaltrials@spurtherapeutics.com
+44 (0)1438 906870
Time Frame
Start Date: 2026-04-07
Estimated Completion Date: 2032-10-01
Participants
Target number of participants: 45
Treatments
Experimental: FLT201
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
Related Therapeutic Areas
Sponsors
Leads: Spur Therapeutics