Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Phase 1

Recruiting

• Conditions: Gaucher Disease, Type 1
• Interventions: Genetic: FLT201

• Registration Number: NCT06545136
• Lead Sponsor: Freeline Therapeutics

Brief Summary

This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-13
• Study First Submitted: 2024-07-29
• Status Verified: 2024-08
• Study First Submitted QC: 2024-08-06
• Last Update Submitted: 2024-08-06
• Study First Posted: 2024-08-09
• Last Update Posted: 2024-08-09
• Primary Completion: 2029-05
• Study Completion: 2029-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

• 1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
• 2. Participants able to give full informed consent and able to comply with all requirements of the trial.

Exclusion Criteria

• n/a

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dosed with FLT201	FLT201	-

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)	Week 38 to Month 60	Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded per CTCAE version 5.0 (or later)

Trial Locations

Locations (4)

Hospital de Clinicas de Porto Alegre (HCPA); 🇧🇷 Porto Alegre, Brazil

Hospital Quironsalud Zaragoza; 🇪🇸 Zaragoza, Spain

Lysosomal Rare Disorders Research and Treatment Center; 🇺🇸 Fairfax, Virginia, United States

Salford Royal Hospital; 🇬🇧 Salford, United Kingdom