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A Gaucher Disease Gene Therapy Trial With FLT201

RecruitingPhase 3Ages 18 Years - anyAllSpur Therapeutics
NCT07223944 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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
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What it tests

  • FLT201
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Where

32 sites
Kaiser Permanente - CA
Los Angeles, United States
UCLA Health
Los Angeles, United States
University of California, San Francisco (UCSF)
San Francisco, United States
University of Colorado Anschutz
Aurora, United States
Emory Healthcare
Atlanta, United States
University of Iowa Health Care
Iowa City, United States
University of Minnesota
Minneapolis, United States
Hackensack University Medical Center
Hackensack, United States

…and 24 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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