Actively Recruiting
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Led by Spur Therapeutics · Updated on 2026-04-21
45
Participants Needed
3
Research Sites
221 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of FLT201 gene therapy in adults with Gaucher disease Type 1 who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years. This Phase 3 study aims to confirm the benefits and safety of FLT201 after patients stop their current treatments. FLT201 is a gene therapy designed to address this rare genetic condition. Participants will receive a single intravenous infusion of FLT201, which is an advanced therapy investigational medicinal product (ATIMP). The therapy uses a special virus vector to deliver genetic material aimed at treating Gaucher disease Type 1. The study is non-randomized and conducted at multiple centers, focusing on adult patients who have maintained stable blood counts and have been on continuous treatment with ERT or SRT for at least two years. During the study, participants will be closely monitored for one year to evaluate the treatment's effects and safety. Researchers will assess the efficacy of FLT201 through various clinical measurements, including blood tests to monitor hemoglobin and platelet levels. Safety evaluations will be ongoing throughout the study. The total duration of participation is at least one year following the gene therapy infusion, with detailed follow-ups to track outcomes and any potential side effects.
CONDITIONS
Brief Title
A Gaucher Disease Gene Therapy Trial With FLT201
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 618 years at time of screening
- Clinical diagnosis of Gaucher disease type 1
- Stable hemoglobin concentration at baseline
- Stable platelet count at baseline
- Receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) without interruption 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 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 year
Participants receive a single intravenous infusion of FLT201 gene therapy.
Multiple visits for monitoring during the 1 year following infusion
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
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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