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A Gaucher Disease Gene Therapy Trial With FLT201
About
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 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.
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
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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Study Stats
Protocol No.
25-1027
Category
Genetic and Rare Diseases
Principal Investigator
Contact
- Sarah Gaunt
Location
- UCLA Westwood