A Gaucher Disease Gene Therapy Trial With FLT201 (GALILEO-3)

April 20, 2026 updated by: Spur Therapeutics

A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

45

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • University of Texas Southwestern
    • Virginia
      • Fairfax, Virginia, United States, 22030-6066
        • Recruiting
        • Lysosomal Rare Disorders Research and Treatment Center
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: FLT201
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion
Genetic: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the efficacy of FLT201
Time Frame: 1 Year
Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52
1 Year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Spur Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 7, 2026

Primary Completion (Estimated)

July 1, 2028

Study Completion (Estimated)

October 1, 2032

Study Registration Dates

First Submitted

October 30, 2025

First Submitted That Met QC Criteria

October 30, 2025

First Posted (Actual)

November 3, 2025

Study Record Updates

Last Update Posted (Actual)

April 21, 2026

Last Update Submitted That Met QC Criteria

April 20, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FLT201-03
  • 2025-520765-50-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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