Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Condition(s) studied
Investigational drug(s) / intervention(s)
Avalglucosidase alfa (GZ402666): Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion
Study summary
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until the conclusion of negotiations of avalglucosidase alfa in France under the standard reimbursement framework on each indication independently, infantile (IOPD) and late (LOPD), or until June 2027, whichever comes first.
\- Study visit frequency: every 2 weeks
Eligibility
Primary outcome measure(s)
- Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death — From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months
Trial sites (11)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
More Genzyme, a Sanofi Company trials in France
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05164055 on ClinicalTrials.gov ↗ ← All trials in France