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)
Status: Active_not_recruiting
Location: See all (4) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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.
Locations
United States
Virginia
Lysosomal Rare Disorders Research and Treatment Center
Fairfax
Other Locations
Brazil
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre
Spain
Hospital Quironsalud Zaragoza
Zaragoza
United Kingdom
Salford Royal Hospital
Salford
Time Frame
Start Date: 2024-05-13
Completion Date: 2029-05
Participants
Target number of participants: 75
Treatments
Experimental: Dosed with FLT201
Related Therapeutic Areas
Sponsors
Leads: Spur Therapeutics