RecruitingPhase 3NCT07223944

A Gaucher Disease Gene Therapy Trial With FLT201

A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1

Sponsor

Spur Therapeutics

Enrollment

45 participants

Start Date

Apr 7, 2026

Study Type

INTERVENTIONAL

Conditions

Gaucher Disease Type 1

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

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying FLT201 for people with gaucher disease type 1. The study is currently recruiting participants at 33 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICFLT201

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.

Locations(33)

Kaiser Permanente - CA

Los Angeles, California, United States

UCLA Health

Los Angeles, California, United States

University of California, San Francisco (UCSF)

San Francisco, California, United States

University of Colorado Anschutz

Aurora, Colorado, United States

Emory Healthcare

Atlanta, Georgia, United States

University of Iowa Health Care

Iowa City, Iowa, United States

University of Minnesota

Minneapolis, Minnesota, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

NYU Langone Health

New York, New York, United States

Columbia University

New York, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

University of Texas Southwestern

Dallas, Texas, United States

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, United States

Hospital Italiano de Buenos Aires

Buenos Aires, Argentina

Hospital Militar Central

Buenos Aires, Argentina

Clinica Universitaria Reina Fabiola

Córdoba, Argentina

Melbourne Health - Royal Melbourne Hospital

Melbourne N., Victoria, Australia

Hospital de Clinicas de Porto Alegre

Porto Alegre, Brazil

MAGIC Clinic Ltd

Calgary, Alberta, Canada

Lady Davis Institute for Medical Research Jewish General Hospital

Montreal, Quebec, Canada

Shaare Zedek Medical Center

Jerusalem, Israel

Rabin Medical Center, Beilinson Hospital

Petah Tikva, Israel

King Faisal Specialist Hospital and Research Center

Riyadh, Saudi Arabia

Hospital Universitario Vall d'Hebron

Barcelona, Barcelona, Spain

Hospital Quironsalud de Zaragoza

Zaragoza, Spain

Gazi University Medical Faculty

Ankara, Yenimahalle, Turkey (Türkiye)

Queen Elizabeth Hospital Birmingham

Birmingham, Birmingham, United Kingdom

Salford Royal Hospital

Salford, Greater Manchester, United Kingdom

Royal Free Hospital

London, London, United Kingdom

Addenbrooke's Hospital

Cambridge, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT07223944

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