A Gaucher Disease Gene Therapy Study (GALILEO-1)
Research type
Research Study
Full title
A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1)
IRAS ID
293417
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Spur Therapeutics Limited
Eudract number
2020-005032-30
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
FLT201, Compound Number
Duration of Study in the UK
2 years, 5 months, 12 days
Research summary
This study is a first-in-human, phase 1/2, 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 the study drug (FLT201). Adult participants who provide consent to participate in this study will be screened for eligibility over a 12 week period.
On the day before the infusion (Day -1), the participant will attend the study site and final eligibility assessments will be completed. On Day 1, the study drug (FLT201) will be administered as a single dose, slow intravenous infusion. Following satisfactory results from safety evaluation done on day 1, the participant will be discharged from the study site.
After the FLT201 dosing, all participants will enter a 38-week follow up period.
Number of participants:
Part 1 (dose escalation): Approximately 9 patients will be enrolled across up to 4 dose cohorts.
Part 2 (dose expansion): Approximately 6 patients will be enrolled across up to 2 dose cohorts.Approximately 15 participants will be enrolled across Part 1 and 2 of the study; the actual number of participants will depend on emerging data.
FLT201 is an in-vivo gene therapy product intended for the treatment of adult patients with Gaucher disease Type 1. It is a recombinant adeno-associated viral vector using the Freeline proprietary capsid S3 (AAVS3) and encodes a protein engineered variant of human beta-glucocerebrosidase (GCase). FLT201 is anticipated to lead to continuous endogenous production of GCase (variant 85) in hepatocytes, resulting in steady plasma activity levels and thus has the potential to provide enhanced uptake of the active enzyme by macrophages and better penetration in the target tissues of Gaucher disease.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0306
Date of REC Opinion
17 Jun 2021
REC opinion
Further Information Favourable Opinion