A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944 | Recruiting Phase 3 FDA Drug FDA Device

• 2 other identifiers: FLT201-032025-520765-50-00
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 3

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

Outcome Measures

Primary Outcomes (1)

• To evaluate the efficacy of FLT201

  Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52

  1 Year

Study Arms (1)

FLT201

EXPERIMENTAL

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

Genetic: FLT201

Interventions

FLT201 GENETIC

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.

FLT201

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Spur Therapeutics: lead

Study Sites (3)

Duke University Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-6066, United States

RECRUITING

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Spur Clinical Trials Contact

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2025
• First Posted: November 3, 2025
• Study Start: April 7, 2026
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): October 1, 2032
• Last Updated: April 21, 2026

Record last verified: 2026-04

Locations

US (3)