Tests FLT201 gene therapy in adults with Gaucher disease type 1
Tests FLT201 gene therapy in adults with Gaucher disease type 1.
Plain-language title generated from the registry record
Registry title: A Gaucher Disease Gene Therapy Trial With FLT201
Key facts
- Where
- Atlanta, Aurora, Cincinnati, Dallas, Durham, Fairfax, Hackensack, Iowa City, Los Angeles, Minneapolis, New York, Philadelphia, Pittsburgh, and San Francisco, United StatesSee the 15 sites
- Phase
- Phase 3
Where in United States
- Emory HealthcareAtlanta, United StatesTaking people
- University of Colorado AnschutzAurora, United StatesTaking people
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesNot yet recruiting
- University of Texas, SouthwesternDallas, United StatesTaking people
- Duke University Medical CenterDurham, United StatesTaking people
- Lysosomal Rare Disorders Research and Treatment CenterFairfax, United StatesTaking people
- Hackensack University Medical CenterHackensack, United StatesTaking people
- University of Iowa Health CareIowa City, United StatesNot yet recruiting
- Kaiser Permanente - CALos Angeles, United StatesTaking people
- UCLA HealthLos Angeles, United StatesTaking people
- University of MinnesotaMinneapolis, United StatesTaking people
- NYU Langone HealthNew York, United StatesTaking people
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
- UPMC Children's Hospital of PittsburghPittsburgh, United StatesTaking people
- University of California, San Francisco (UCSF)San Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Spur Therapeutics
- Trial code
- NCT07223944EU 2025-520765-50-00
- Record updated
- 14 October 2026
- Original 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.Read full original descriptionShow less
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
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.
Inclusion: 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.
Exclusion: Positive for AAVS3 neutralizing antibodies; History of receiving any gene therapy or cell therapy; Diagnosed or suspected Gaucher disease type 2 or type 3; History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant; History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply; Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study; Positive pregnancy test or lactating.