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NCT07223944PHASE3Recruiting

A Gaucher Disease Gene Therapy Trial With FLT201

Spur Therapeutics

Start Date

4/7/2026

Completion Date

10/1/2032

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 Criteria

Age Range: 18 years to No maximum

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

Interventions

GENETIC

FLT201

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Conditions

Gaucher Disease Type 1

Locations

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