Recruiting

Phase 3

FLT201A Gaucher Disease Gene Therapy Trial With FLT201

Sponsor:

Spur Therapeutics

Code:

NCT07223944

Conditions

Gaucher Disease Type 1

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

FLT201

Study Details

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.

Conditions

Gaucher Disease Type 1

Study ID

NCT07223944

Start date

Apr 7, 2026

Status verified date

Apr, 2026

Completion date

Oct 1, 2032

Anticipated

Primary completion date

Jul 1, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

45 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: FLT201

FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion

Interventions

FLT201

FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.

Primary outcome measure

To evaluate the efficacy of FLT201 [ Time Frame: 1 Year ]

Central Contacts and Locations

Central contacts

Spur Clinical Trials Contact

+44 (0)1438 906870 clinicaltrials@spurtherapeutics.com

Locations

Kaiser Permanente - CA

Recruiting

Los Angeles, California, United States, 90027

Contacts

Dr. Divya Vats

626-646-4612 divya.vats@kp.org

UCLA Health

Recruiting

Los Angeles, California, United States, 90095

Contacts

Dr. Nicola Longo

310-825-5610 wsia@mednet.ucla.edu

University of California, San Francisco (UCSF)

Recruiting

San Francisco, California, United States, 94609

Contacts

Dr. Irene Chang

510-428-3885 Irene.chang@ucsf.edu

University of Colorado Anschutz

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Dr. Matthew Taylor

720-446-6680 matthew.taylor@CUAnschutz.edu

Emory Healthcare

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Dr. William Wilcox

404-778-8618 william.wilcox@emory.edu

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55454

Contacts

Dr. Whitley

612-625-1594 whitley@umn.edu

Brenda Diethelm-Okita

dieth001@umn.edu

NYU Langone Health

Recruiting

New York, New York, United States, 10017

Contacts

Dr. Nicolas Abreu

929-455-5629 Neurogenetics@nyulangone.org

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Dr. Priya Kishnani

919-215-0097 priya.kishnani@duke.edu

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Dr. Can Ficicioglu

215-590-3376 FICICIOGLU@chop.edu

UPMC Children's Hospital of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Dr. Damara Ortiz

412-692-3475 Nadene.Henderson@chp.edu

University of Texas Southwestern

Recruiting

Dallas, Texas, United States, 75390

Contacts

Dr. Markey McNutt

214-648-1222 geneticsresearch@utsouthwestern.edu

Jordan Roberts

jordan.roberts2@UTSouthwestern.edu

Lysosomal Rare Disorders Research and Treatment Center

Recruiting

Fairfax, Virginia, United States, 22030-6066

Contacts

Lauren Noll

(703) 2616220 lnoll@ldrtc.org

Ozlem Goker-Alpan

(703) 2616220 ogoker-alpan@ldrtc.org

MAGIC Clinic Ltd

Recruiting

Calgary, Alberta, Canada, T2E 7H7

Contacts

Dr. Aneal Khan

587-885-3158 research@magiccalgary.ca

Lady Davis Institute for Medical Research Jewish General Hospital

Recruiting

Montreal, Quebec, Canada, T2E 7Z4

More Information

Sponsor

Spur Therapeutics

Last update posted

Sep 3, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Spur Therapeutics on 2026-09-03.