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Clinical Trials in Spain / NCT07223944
Recruiting Phase 3

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944 · tracked via the Priya Life Science Spain tracker
Sponsor
Spur Therapeutics
Phase
Phase 3
Started
2026-04-07
Last updated
2026-09-03

Condition(s) studied

Gaucher Disease Type 1

Investigational drug(s) / intervention(s)

FLT201

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.

Study 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

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria: * 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 Key Exclusion Criteria: * 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. * History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply

Primary outcome measure(s)

  • To evaluate the efficacy of FLT201 — 1 Year
    Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52

Trial sites (33)

FacilityCityRegionStatus
Kaiser Permanente - CA Los Angeles California Recruiting
UCLA Health Los Angeles California Recruiting
University of California, San Francisco (UCSF) San Francisco California Recruiting
University of Colorado Anschutz Aurora Colorado Recruiting
Emory Healthcare Atlanta Georgia Recruiting
University of Iowa Health Care Iowa City Iowa Not Yet Recruiting
University of Minnesota Minneapolis Minnesota Recruiting
Hackensack University Medical Center Hackensack New Jersey Not Yet Recruiting
NYU Langone Health New York New York Recruiting
Columbia University New York New York Not Yet Recruiting
Duke University Medical Center Durham North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Not Yet Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania Recruiting
University of Texas Southwestern Dallas Texas Recruiting
Lysosomal Rare Disorders Research and Treatment Center Fairfax Virginia Recruiting
Hospital Italiano de Buenos Aires Buenos Aires Argentina Recruiting
Hospital Militar Central Buenos Aires Argentina Recruiting
Clinica Universitaria Reina Fabiola Córdoba Argentina Recruiting
Melbourne Health - Royal Melbourne Hospital Melbourne N. Victoria Not Yet Recruiting
Hospital de Clinicas de Porto Alegre Porto Alegre Brazil Recruiting
MAGIC Clinic Ltd Calgary Alberta Recruiting
Lady Davis Institute for Medical Research Jewish General Hospital Montreal Quebec Recruiting
Shaare Zedek Medical Center Jerusalem Israel Recruiting
Rabin Medical Center, Beilinson Hospital Petah Tikva Israel Recruiting
King Faisal Specialist Hospital and Research Center Riyadh Saudi Arabia Not Yet Recruiting
Hospital Universitario Vall d'Hebron Barcelona Barcelona Not Yet Recruiting
Hospital Quironsalud de Zaragoza Zaragoza Spain Not Yet Recruiting
Gazi University Medical Faculty Ankara Yenimahalle Not Yet Recruiting
Queen Elizabeth Hospital Birmingham Birmingham Birmingham Not Yet Recruiting
Salford Royal Hospital Salford Greater Manchester Not Yet Recruiting
Royal Free Hospital London London Recruiting
Addenbrooke's Hospital Cambridge United Kingdom Not Yet Recruiting

More Spur Therapeutics trials in Spain

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07223944 on ClinicalTrials.gov ↗ ← All trials in Spain