Pompe disease patients get continued enzyme therapy in Long-Term safety study
NCT ID NCT05164055
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Investigational Site Number : 2500001
Marseille, 13385, France
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Investigational Site Number : 2500002
Paris, 75013, France
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Investigational Site Number : 2500003
Lyon, 69003, France
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Investigational Site Number : 2500004
Bordeaux, 33000, France
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Investigational Site Number : 2500005
Brest, 29609, France
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Investigational Site Number : 2500006
Nantes, 44093, France
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Investigational Site Number : 2500007
Nice, 06200, France
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Investigational Site Number : 2500008
Clermont-Ferrand, 63000, France
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Investigational Site Number : 2500009
Lille, 59037, France
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Investigational Site Number : 2500010
Paris, 75015, France
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Investigational Site Number : 2500011
Tours, 37044, France