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ACTIVE NOT RECRUITING
NCT05164055
PHASE4

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

Sponsor: Genzyme, a Sanofi Company

View on ClinicalTrials.gov

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

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

Key Details

Gender

All

Age Range

6 Months - Any

Study Type

INTERVENTIONAL

Enrollment

17

Start Date

2022-07-11

Completion Date

2026-09-30

Last Updated

2025-10-31

Healthy Volunteers

No

Interventions

DRUG

Avalglucosidase alfa (GZ402666)

Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Locations (11)

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