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

NCT06545136 · tracked via the Priya Life Science UK tracker

Sponsor

Spur Therapeutics

Phase

Phase 1/2

Started

2024-05-13

Last updated

2025-11-04

Condition(s) studied

Gaucher Disease, Type 1

Investigational drug(s) / intervention(s)

FLT201

FLT201: 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.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

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 sites (4)

Facility	City	Region
Lysosomal Rare Disorders Research and Treatment Center	Fairfax	Virginia
Hospital de Clinicas de Porto Alegre (HCPA)	Porto Alegre	Brazil
Hospital Quironsalud Zaragoza	Zaragoza	Spain
Salford Royal Hospital	Salford	United Kingdom

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06545136 on ClinicalTrials.gov ↗ ← All trials in the UK