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
RECRUITING
Los Angeles
UCLA Health
RECRUITING
Los Angeles
University of California, San Francisco (UCSF)
RECRUITING
San Francisco
Colorado
University of Colorado Anschutz
RECRUITING
Aurora
Georgia
Emory Healthcare
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
RECRUITING
New York
Ohio
Cincinnati Children's Hospital Medical Center
NOT_YET_RECRUITING
Cincinnati
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
UPMC Children's Hospital of Pittsburgh
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
RECRUITING
Buenos Aires
Hospital Militar Central
RECRUITING
Buenos Aires
Clinica Universitaria Reina Fabiola
RECRUITING
Córdoba
Australia
Melbourne Health - Royal Melbourne Hospital
NOT_YET_RECRUITING
Melbourne N.
Brazil
Hospital de Clinicas de Porto Alegre
RECRUITING
Porto Alegre
Canada
MAGIC Clinic Ltd
RECRUITING
Calgary
Lady Davis Institute for Medical Research Jewish General Hospital
RECRUITING
Montreal
Israel
Shaare Zedek Medical Center
RECRUITING
Jerusalem
Rabin Medical Center, Beilinson Hospital
RECRUITING
Petah Tikva
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 Hospital
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