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Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Sponsor: Genzyme, a Sanofi Company
Summary
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to enzyme replacement therapy (ERT) with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first. \- Study visit frequency: every 2 weeks
Official title: A French Multicenter Open Label Phase 4 Extension Study of Long-term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Alfa Development Studies in France
Key Details
Gender
All
Age Range
6 Months - Any
Study Type
INTERVENTIONAL
Enrollment
17
Start Date
2022-07-11
Completion Date
2026-09-30
Last Updated
2025-10-31
Healthy Volunteers
No
Conditions
Interventions
Avalglucosidase alfa (GZ402666)
Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion
Locations (11)
Investigational Site Number : 2500004
Bordeaux, France
Investigational Site Number : 2500005
Brest, France
Investigational Site Number : 2500008
Clermont-Ferrand, France
Investigational Site Number : 2500009
Lille, France
Investigational Site Number : 2500003
Lyon, France
Investigational Site Number : 2500001
Marseille, France
Investigational Site Number : 2500006
Nantes, France
Investigational Site Number : 2500007
Nice, France
Investigational Site Number : 2500002
Paris, France
Investigational Site Number : 2500010
Paris, France
Investigational Site Number : 2500011
Tours, France