FLT201 gene therapy for adults with Gaucher disease type 1

A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1

This trial tests a one-time gene therapy called FLT201 in adults with Gaucher disease type 1 who have been on stable enzyme replacement or substrate reduction therapy to see if they can stop those treatments and keep their blood counts and disease control stable.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 45 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Spur Therapeutics (industry)
• Locations: 3 sites (Durham, North Carolina and 2 other locations)
• Trial ID: NCT07223944 on ClinicalTrials.gov

What this trial studies

This is a Phase 3, non-randomized, multicenter study enrolling adults with Gaucher disease type 1 who have been on uninterrupted enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years. Eligible participants will receive a single administration of FLT201 and then discontinue their prior ERT/SRT while being closely followed for efficacy and safety outcomes. Key safety exclusions include positive AAVS3 neutralizing antibodies, prior gene/cell therapy, prior hematopoietic stem cell or solid organ transplant, and total splenectomy. The trial is sponsored by Spur Therapeutics and is being conducted at several major U.S. lysosomal-disease centers.

Who should consider this trial

Good fit: Adults (≥18 years) with a clinical diagnosis of Gaucher disease type 1 who have received uninterrupted ERT or SRT for at least two years and have stable hemoglobin and platelet counts are the intended participants.

Not a fit: People with neuronopathic Gaucher (types 2 or 3), positive AAVS3 neutralizing antibodies, prior gene or cell therapy, prior HSCT or total splenectomy, pregnant or lactating individuals, or those with disqualifying laboratory abnormalities or medical conditions are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, FLT201 could allow adults with Gaucher type 1 to stop regular enzyme replacement or substrate reduction therapy while maintaining stable blood counts and organ function.

How similar studies have performed: Early-phase AAV-based gene therapy efforts for Gaucher and other lysosomal storage disorders have shown promising signals in safety and biological activity, so this Phase 3 is a confirmatory step for a relatively novel approach.

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

Where this trial is running

Durham, North Carolina and 2 other locations

• Duke University Medical Center — Durham, North Carolina, United States (RECRUITING)
• University of Texas Southwestern — Dallas, Texas, United States (RECRUITING)
• Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States (RECRUITING)

Study contacts

• Study coordinator: Spur Clinical Trials Contact

Conditions: Gaucher Disease Type 1