Status:
NOT_YET_RECRUITING
A Gaucher Disease Gene Therapy Trial With FLT201
Lead Sponsor:
Spur Therapeutics
Conditions:
Gaucher Disease Type 1
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief 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 re...
Eligibility Criteria
Inclusion
- Key
- 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
- 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
Key Trial Info
Start Date :
July 1 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2032
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT07223944
Start Date
July 1 2026
End Date
October 1 2032
Last Update
November 3 2025
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