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A Gene Therapy Study in Patients With Gaucher Disease Type 1 — GALILEO-1  

Gaucher Disease, Type 1 Clinical Trial

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

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05324943
Study type Interventional
Source Freeline Therapeutics
Contact Clinical Operations
Phone +44 1438 906870
Email contact@freeline.life
Status Recruiting
Phase Phase 1
Start date April 15, 2022
Completion date January 31, 2025
View Details
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