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A Gaucher Disease Gene Therapy Trial With FLT201
A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944

RecruitingPhase 3

Sponsor: Spur Therapeutics

Conditions: Gaucher Disease Type 1

Interventions: FLT201

Countries: United States, Argentina, Australia, Brazil, Canada, Israel, Saudi Arabia, Spain

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 overview

Sex: ALL

Age: 18 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

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
Locations (33)
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • San Francisco, California, United States
  • Aurora, Colorado, United States
  • Atlanta, Georgia, United States
  • Iowa City, Iowa, United States
  • Minneapolis, Minnesota, United States
  • Hackensack, New Jersey, United States
  • New York, New York, United States
  • New York, New York, United States
  • Durham, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Philadelphia, Pennsylvania, United States
  • Pittsburgh, Pennsylvania, United States
  • Dallas, Texas, United States
  • Fairfax, Virginia, United States
  • Buenos Aires, Argentina
  • Buenos Aires, Argentina
  • Córdoba, Argentina
  • Melbourne N., Victoria, Australia
  • Porto Alegre, Brazil
  • Calgary, Alberta, Canada
  • Montreal, Quebec, Canada
  • Jerusalem, Israel
  • Petah Tikva, Israel
  • Riyadh, Saudi Arabia
  • Barcelona, Barcelona, Spain
  • Zaragoza, Spain
  • Ankara, Yenimahalle, Turkey (Türkiye)
  • Birmingham, Birmingham, United Kingdom
  • + 3 more on CT.gov