French patients continue Life-Changing pompe treatment in Long-Term safety study

NCT ID NCT05164055

Ongoing Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

Summary

This study provides continued access to enzyme replacement therapy for French patients with Pompe disease who completed earlier research trials. It follows 17 patients for approximately four years to monitor long-term safety and track how well the treatment maintains their walking ability, breathing function, and quality of life. The goal is to gather more evidence about this treatment's ongoing benefits and risks until it becomes routinely available through France's healthcare system.

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Contacts and locations

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Locations

Investigational Site Number : 2500001

Marseille, 13385, France
Investigational Site Number : 2500002

Paris, 75013, France
Investigational Site Number : 2500003

Lyon, 69003, France
Investigational Site Number : 2500004

Bordeaux, 33000, France
Investigational Site Number : 2500005

Brest, 29609, France
Investigational Site Number : 2500006

Nantes, 44093, France
Investigational Site Number : 2500007

Nice, 06200, France
Investigational Site Number : 2500008

Clermont-Ferrand, 63000, France
Investigational Site Number : 2500009

Lille, 59037, France
Investigational Site Number : 2500010

Paris, 75015, France
Investigational Site Number : 2500011

Tours, 37044, France

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE II