A Gaucher Disease Gene Therapy Trial With FLT201

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Gaucher Disease Type 1 Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Gaucher Disease Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

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

Study contacts

Contact information is provided by the study sponsor or research team.

Spur Clinical Trials Contact

CONTACT

[email protected]

+44 (0)1438 906870

Sponsors and collaborators

Lead sponsor

Spur Therapeutics

Industry

Registry information

Official study title

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

Acronym: GALILEO-3

Important dates

Study start: 2026
Primary completion: 2028
Study completion: 2032
First posted: Nov 3, 2025
Registry last updated: Jul 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Recruiting