A Gaucher Disease Gene Therapy Trial With FLT201
GALILEO-3
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
2 other identifiers
interventional
45
10 countries
33
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Apr 2026
Longer than P75 for phase_3
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 30, 2025
CompletedFirst Posted
Study publicly available on registry
November 3, 2025
CompletedStudy Start
First participant enrolled
April 7, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 1, 2032
July 13, 2026
April 1, 2026
2.2 years
October 30, 2025
July 10, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
To evaluate the efficacy of FLT201
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
1 Year
Study Arms (1)
FLT201
EXPERIMENTALFLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Diagnosed or suspected Gaucher disease type 2 or type 3
- Positive for AAVS3 neutralizing antibodies.
- Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- Positive pregnancy test or lactating
- History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.
- History of receiving any gene therapy or cell therapy.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (33)
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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 30, 2025
First Posted
November 3, 2025
Study Start
April 7, 2026
Primary Completion (Estimated)
July 1, 2028
Study Completion (Estimated)
October 1, 2032
Last Updated
July 13, 2026
Record last verified: 2026-04