FLT201 gene therapy for adults with Gaucher disease type 1

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Kaiser Permanente - CA
  Los Angeles, California, United States Recruiting
• UCLA Health
  Los Angeles, California, United States Recruiting
• University of California, San Francisco (UCSF)
  San Francisco, California, United States Recruiting
• University of Colorado Anschutz
  Aurora, Colorado, United States Recruiting
• Emory Healthcare
  Atlanta, Georgia, United States Recruiting
• University of Minnesota
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Study coordinator: Spur Clinical Trials Contact
• Email: clinicaltrials@spurtherapeutics.com
• Phone: +44 (0)1438 906870

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.