clinical trial · NCT05324943
A Gene Therapy Study in Patients With Gaucher Disease Type 1
Gaucher Disease, Type 1FLT201
brief 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.
started
Apr 15, 2022
primary completion
Dec 4, 2024
completion
Dec 4, 2024
last updated
Nov 4, 2025
official title
A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol