New hope for babies with pompe disease who are failing standard therapy
NCT ID NCT03019406
First seen Jan 12, 2026 · Last updated May 16, 2026 · Updated 19 times
Summary
This study tests a new enzyme therapy called avalglucosidase alfa in 22 children with infantile-onset Pompe disease who are not doing well on the current standard treatment. The goal is to see if the new drug is safe and can better control the disease. Participants receive increasing doses of the study drug through an IV.
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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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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
Conditions
Explore the condition pages connected to this study.