Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

October 31, 2025 updated by: Spur Therapeutics

A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects With Gaucher Disease (GALILEO-2)

This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

75

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

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

Study Locations

  • Brazil
      • Porto Alegre, Brazil
        • Hospital de Clinicas de Porto Alegre (HCPA)
  • Spain
      • Zaragoza, Spain
        • Hospital Quironsalud Zaragoza
  • United Kingdom
      • Salford, United Kingdom
        • Salford Royal Hospital
  • United States
    • Virginia
      • Fairfax, Virginia, United States, 22030-6066
        • Lysosomal Rare Disorders Research and Treatment Center

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

Inclusion Criteria:

  • 1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
  • 2. Participants able to give full informed consent and able to comply with all requirements of the trial.

Exclusion Criteria:

  • n/a

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: Dosed with FLT201
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
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
Time Frame: Week 38 to Month 60
Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded per CTCAE version 5.0 (or later)
Week 38 to Month 60

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)

May 13, 2024

Primary Completion (Estimated)

May 1, 2029

Study Completion (Estimated)

May 1, 2029

Study Registration Dates

First Submitted

July 29, 2024

First Submitted That Met QC Criteria

August 6, 2024

First Posted (Actual)

August 9, 2024

Study Record Updates

Last Update Posted (Estimated)

November 4, 2025

Last Update Submitted That Met QC Criteria

October 31, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FLT201-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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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