NCT05164055

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

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

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

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

Detailed description

Treatment duration approximately 4 years and 3 months: until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
DRUG	Avalglucosidase alfa (GZ402666)	Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Timeline

• Start date: 2022-07-11
• Primary completion: 2026-09-30
• Completion: 2026-09-30
• First posted: 2021-12-20
• Last updated: 2025-10-31

Locations

11 sites across 1 country: France