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RECRUITING Phase 3 ClinicalTrials.gov

A Gaucher Disease Gene Therapy Trial With FLT201

Sponsor: Spur Therapeutics • ClinicalTrials.gov ID: NCT07223944

Registry updated: 2026-10-14
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Description

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.

Interventions

  • FLT201 (Genetic)
    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

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

Conditions

Gaucher Disease Type 1

Locations

  • 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
  • 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
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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record