New hope for babies battling rare Muscle-Wasting disease
NCT ID NCT03019406
Summary
This study is testing a new enzyme replacement drug called avalglucosidase alfa for children under 18 with severe infantile-onset Pompe disease. It's for children who are already receiving the current standard treatment but are still getting worse or not responding well enough. The main goals are to see if the new drug is safe and if it can help improve muscle function, heart health, and breathing.
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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.