Actively Recruiting
A Gaucher Disease Gene Therapy Trial With FLT201
Led by Spur Therapeutics · Updated on 2026-04-21
45
Participants Needed
3
Research Sites
338 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Gaucher Disease Gene Therapy Trial With FLT201
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 18 years or older at screening
- Clinical diagnosis of Gaucher disease Type 1
- Stable hemoglobin concentration at baseline
- Stable platelet count at baseline
- Receiving enzyme replacement therapy or substrate reduction therapy continuously for at least 2 years
You will not qualify if you...
- Diagnosed or suspected Gaucher disease Type 2 or Type 3
- Positive for AAVS3 neutralizing antibodies
- Abnormal lab values, conditions, or diseases making participation unsuitable
- Positive pregnancy test or currently lactating
- History of hematopoietic stem cell transplant, bone marrow transplant, or any solid organ transplant
- History of receiving gene therapy or cell therapy
- History of total splenectomy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
2
University of Texas Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
3
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030-6066
Actively Recruiting
Research Team
S
Spur Clinical Trials Contact
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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