NCT07223944 A Gaucher Disease Gene Therapy Trial With FLT201
| NCT ID | NCT07223944 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Spur Therapeutics |
| Condition | Gaucher Disease Type 1 |
| Study Type | INTERVENTIONAL |
| Enrollment | 45 participants |
| Start Date | 2026-04-07 |
| Primary Completion | 2028-07-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 45 participants in total. It began in 2026-04-07 with a primary completion date of 2028-07-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07223944 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Gaucher Disease Type 1. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07223944 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 45 participants.
Is NCT07223944 currently recruiting?
Yes, NCT07223944 is actively recruiting participants. Contact the research team at clinicaltrials@spurtherapeutics.com for enrollment information.
Where is the NCT07223944 trial being conducted?
This trial is being conducted at Los Angeles, United States, Los Angeles, United States, San Francisco, United States, Aurora, United States and 11 additional locations.
Who is sponsoring the NCT07223944 clinical trial?
NCT07223944 is sponsored by Spur Therapeutics. The trial plans to enroll 45 participants.