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Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
Sponsor: Spur Therapeutics
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.
Official title: A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects With Gaucher Disease (GALILEO-2)
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
75
Start Date
2024-05-13
Completion Date
2029-05
Last Updated
2025-11-04
Healthy Volunteers
No
Conditions
Interventions
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.
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