Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Status:
Not yet recruiting
Trial end date:
2023-05-31
Target enrollment:
Participant gender:
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 January
2023, whichever comes first.
- Study visit frequency: every 2 weeks