Clinical Trial

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

Active, Not Recruiting Phase 4
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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
Protocol Amendment History 14 amendments
This ClinicalTrials.gov record has been amended 14 times since 2021-12-07; most recent amendment 2025-10-29.
Status change: Recruiting → Active, Not Recruiting 2023-08-04
Status change: Not Yet Recruiting → Recruiting 2022-06-23
Trial Details
NCT Number NCT05164055
Lead Sponsor Genzyme, a Sanofi Company
Conditions Glycogen Storage Disease Type II
Enrollment 17 participants
Start Date 2022-07-11
Primary Completion 2026-09-30 (estimated)
Study Completion 2026-09-30 (estimated)
Updated on ClinicalTrials.gov 2025-10-31