A Gaucher Disease Gene Therapy Trial With FLT201
Spur Therapeutics · NCT07223944
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 18+ · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 3
- Target enrollment
- 45
- US sites recruiting
- 13
- Study start
- April 7, 2026
- Record last updated
- October 14, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study 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.
Being studied: FLT201
Conditions: Gaucher Disease Type 1
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full 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
Recruiting US locations
Kaiser Permanente - CA
Los Angeles, CA 90027
Site contact: Dr. Divya Vats · 626-646-4612 · divya.vats@kp.org
UCLA Health
Los Angeles, CA 90095
Site contact: Dr. Nicola Longo · 310-825-5610 · wsia@mednet.ucla.edu
University of California, San Francisco (UCSF)
San Francisco, CA 94609
Site contact: Dr. Irene Chang · 510-428-3885 · Irene.chang@ucsf.edu
University of Colorado Anschutz
Aurora, CO 80045
Site contact: Dr. Matthew Taylor · 720-446-6680 · matthew.taylor@CUAnschutz.edu
Emory Healthcare
Atlanta, GA 30322
Site contact: Dr. William Wilcox · 404-778-8618 · william.wilcox@emory.edu
University of Minnesota
Minneapolis, MN 55454
Site contact: Dr. Whitley · 612-625-1594 · whitley@umn.edu
Hackensack University Medical Center
Hackensack, NJ 07601
Site contact: Dr. Helio Pedro · 551-996-8178 · helio.pedro@hmhn.org
NYU Langone Health
New York, NY 10017
Site contact: Dr. Nicolas Abreu · 929-455-5629 · Neurogenetics@nyulangone.org
Duke University Medical Center
Durham, NC 27710
Site contact: Dr. Priya Kishnani · 919-215-0097 · priya.kishnani@duke.edu
Children's Hospital of Philadelphia
Philadelphia, PA 19104
Site contact: Dr. Can Ficicioglu · 215-590-3376 · FICICIOGLU@chop.edu
UPMC Children's Hospital of Pittsburgh
Pittsburgh, PA 15224
Site contact: Dr. Damara Ortiz · 412-692-3475 · Nadene.Henderson@chp.edu
University of Texas Southwestern
Dallas, TX 75390
Site contact: Dr. Markey McNutt · 214-648-1222 · geneticsresearch@utsouthwestern.edu
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, VA 22030-6066
Site contact: Lauren Noll · (703) 2616220 · lnoll@ldrtc.org
Study contacts (from the official record)
- Spur Clinical Trials Contact · +44 (0)1438 906870 · clinicaltrials@spurtherapeutics.com