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Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Glycogen Storage Disease Type II Clinical Trial

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

Clinical Trial 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 December 2024, whichever comes first. - Study visit frequency: every 2 weeks

Clinical Trial Description

Treatment duration approximately 2 years and 6 months: until reimbursement of avalglucosidase alfa in France or until December 2024, whichever comes first ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05164055
Study type Interventional
Source Sanofi
Contact
Status Active, not recruiting
Phase Phase 4
Start date July 11, 2022
Completion date December 31, 2024
View Details
See also
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