CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 10 enrolled / 10 target
Drug / intervention
FLT201genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT05324943
NCT05324943Phase 1CompletedUpdate Overdue (0.2/mo)Completion was 18mo ago
CompareCT.gov

A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)

Spur Therapeutics·interventional·Posted Apr 13, 2022·Updated Jun 2, 2026

In Brief

A Phase 1 clinical trial evaluating FLT201 for Gaucher Disease, Type 1. Completed, enrolled 10 participants across 10 sites in 6 countries.

Signals

Enrollment appears stalled

Detailed Summary

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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGaucher Disease, Type 1
CountriesBrazil, Germany, Israel, Spain, United Kingdom, United States
Collaborators--

Timeline

Phase 1CompletedFinished
2023202420252026
First PostedApr 13, 2022
Enrollment StartApr 15, 2022
Primary CompletionDec 4, 2024
TodayJul 2, 2026
Enrollment to primary: 2.6 yearsPosted 4.2 years ago

Arms & Interventions

FLT201 (4.5 × 10^11 vg/kg)experimental

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

Genetic: FLT201

Interventions

FLT201genetic

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.