NCT05324943: GALILEO-1

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Completed Phase 1 Last updated 4 November 2025

What this trial tests

Phase 1 trial testing FLT201 in Gaucher Disease, Type 1 in 6 participants. Completed in 4 December 2024.

Timeline

15 April 2022

Primary endpoint
4 December 2024

4 December 2024

Quick facts

Lead sponsor	Spur Therapeutics
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	6
Start date	15 April 2022
Primary completion	4 December 2024
Estimated completion	4 December 2024
Sites	10 locations across Brazil, United Kingdom, Israel, Germany, United States, Spain

Drugs / interventions tested

• FLT201

Conditions studied

• Gaucher Disease, Type 1 — all drugs for Gaucher Disease, Type 1 →

Sponsor

Spur Therapeutics

Who can join

18 and older, any sex, with Gaucher Disease, Type 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis.
   Shen W, Liu S, Ou L. · · 2022 · cited 123× · PMID 36389770 · DOI 10.3389/fimmu.2022.1001263
2. Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders.
   Daci R, Flotte TR. · · 2024 · cited 39× · PMID 38256124 · DOI 10.3390/ijms25021050
3. AAV Engineering for Improving Tropism to the Central Nervous System.
   Ghauri MS, Ou L. · · 2023 · cited 37× · PMID 36829465 · DOI 10.3390/biology12020186
4. Gene Therapy of Sphingolipid Metabolic Disorders.
   Shaimardanova AA, Solovyeva VV, Issa SS, Rizvanov AA. · · 2023 · cited 18× · PMID 36835039 · DOI 10.3390/ijms24043627
5. Current developments of gene therapy in human diseases.
   Liu F, Li R, Zhu Z, Yang Y, et al · · 2024 · cited 17× · PMID 39156766 · DOI 10.1002/mco2.645
6. The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy.
   Puzzo F, Kay MA. · · 2025 · cited 15× · PMID 40156191 · DOI 10.1016/j.ymthe.2025.03.041
7. Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases.
   Sussman C, Liberatore RA, Drozdz MM. · · 2024 · cited 13× · PMID 38675196 · DOI 10.3390/pharmaceutics16040535
8. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach.
   Feng S, Rcheulishvili N, Jiang X, Zhu P, et al · · 2024 · cited 12× · PMID 38617529 · DOI 10.7150/ijbs.87741