New hope for kids with pompe disease: experimental drug shows promise
NCT ID NCT03019406
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests a new enzyme replacement therapy called avalglucosidase alfa (Nexviazyme) in 22 children with infantile-onset Pompe disease who are not responding well to standard treatment. The study aims to see if the new drug is safe and can improve outcomes. Participants receive the drug through an IV infusion, and researchers monitor for side effects and immune responses.
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 (Nexviazyme)
What this could lead to
If it works, this could offer a better treatment option for children with Pompe disease who are not doing well on current enzyme replacement therapy.
What could go wrong
This is a small, early-phase trial with only 22 participants, so results may not apply to all patients. The drug may cause side effects like infusion reactions.
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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Duke University Medical Center Site Number : 8400001
Durham, North Carolina, 27710-4000, United States
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Investigational Site Number : 1580001
Hsinchu, 30059, Taiwan
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Investigational Site Number : 2500001
Tours, 37044, France
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Investigational Site Number : 2500002
Paris, 75019, France
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Investigational Site Number : 2500003
Paris, 75015, France
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Investigational Site Number : 2500004
Nantes, 44093, France
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Investigational Site Number : 3920001
Fuchu-shi, Tokyo, 183-0042, Japan
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Investigational Site Number : 3920002
Fuchu-shi, Tokyo, 183-8561, Japan
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Investigational Site Number : 8260001
London, London, City of, WC1N 3JH, United Kingdom
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Investigational Site Number : 8260002
Manchester, M13 9WL, United Kingdom
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Regional Medical Genetics Center of New York Site Number : 8400002
Valhalla, New York, 10595, United States
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Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005
Seattle, Washington, 98040, United States