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

Phase 2Active Not RecruitingNCT06545136

Spur Therapeutics75 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Gaucher Disease, Type 1

Interventions

FLT201GENETIC

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.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (4)

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, United States

Hospital de Clinicas de Porto Alegre (HCPA)

Porto Alegre, Brazil

Hospital Quironsalud Zaragoza

Zaragoza, Spain

Salford Royal Hospital

Salford, United Kingdom

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)

Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded per CTCAE version 5.0 (or later)

Time frame: Week 38 to Month 60

Data from ClinicalTrials.gov

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