New enzyme therapy gives hope to babies with rare muscle disease
NCT ID NCT04910776
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. The main goal is to see if it helps babies survive and stay off breathing machines.
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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
Locations
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Advanced Medical Genetics- Site Number : 8400002
Hawthorne, New York, 10532, United States
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Children's Hospitals and Clinics of Minnesota- Site Number : 8400008
Minneapolis, Minnesota, 55404, United States
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Cincinnati Children's Hospital Medical Center- Site Number : 8400001
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center- Site Number : 8400004
Durham, North Carolina, 27710, United States
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Investigational Site Number : 0560001
Leuven, 3000, Belgium
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Investigational Site Number : 1560001
Shanghai, 200120, China
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Investigational Site Number : 1560002
Qingdao, 266034, China
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Investigational Site Number : 1580001
Taipei, 100, Taiwan
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Investigational Site Number : 2760001
Bad Oeynhausen, 35392, Germany
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Investigational Site Number : 3800002
Monza, Lombardy, 20052, Italy
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Investigational Site Number : 5280001
Rotterdam, 3015 CE, Netherlands
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Investigational Site Number : 7240001
Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain
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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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Seattle Children's Hospital- Site Number : 8400003
Seattle, Washington, 98105, United States
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Stanford Hospital- Site Number : 8400006
Stanford, California, 94305, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can early enzyme therapy help babies with pompe disease breathe on their own?
- New hope for babies with rare muscle disease: enzyme therapy trial launches in china
- New hope for kids with pompe disease: experimental drug shows promise
- Pompe disease patients invited to join worldwide registry
- Global pompe registry aims to unlock secrets of rare disease
- Pompe disease patients get continued enzyme therapy in Long-Term safety study