Pompe disease patients get continued enzyme therapy in Long-Term safety study

NCT ID NCT05164055

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the drug is covered by French health insurance or until September 2026.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Avalglucosidase alfa (Nexviadyme), an enzyme replacement therapy given by IV infusion every 2 weeks
What this could lead to: If successful, this study could confirm that long-term use of avalglucosidase alfa is safe and helps manage Pompe disease symptoms.
What could go wrong: This is a small, open-label extension study with only 17 participants, so results may not apply to everyone. Side effects like infusion reactions are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GLYCOGEN STORAGE DISEASE TYPE II

Contacts and locations

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

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