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.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GLYCOGEN STORAGE DISEASE TYPE II are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Advanced Medical Genetics- Site Number : 8400002
Hawthorne, New York, 10532, United States
-
Children's Hospitals and Clinics of Minnesota- Site Number : 8400008
Minneapolis, Minnesota, 55404, United States
-
Cincinnati Children's Hospital Medical Center- Site Number : 8400001
Cincinnati, Ohio, 45229, United States
-
Duke University Medical Center- Site Number : 8400004
Durham, North Carolina, 27710, United States
-
Investigational Site Number : 0560001
Leuven, 3000, Belgium
-
Investigational Site Number : 1560001
Shanghai, 200120, China
-
Investigational Site Number : 1560002
Qingdao, 266034, China
-
Investigational Site Number : 1580001
Taipei, 100, Taiwan
-
Investigational Site Number : 2760001
Bad Oeynhausen, 35392, Germany
-
Investigational Site Number : 3800002
Monza, Lombardy, 20052, Italy
-
Investigational Site Number : 5280001
Rotterdam, 3015 CE, Netherlands
-
Investigational Site Number : 7240001
Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain
-
Investigational Site Number : 8260001
London, London, City of, WC1N 3JH, United Kingdom
-
Investigational Site Number : 8260002
Manchester, M13 9WL, United Kingdom
-
Seattle Children's Hospital- Site Number : 8400003
Seattle, Washington, 98105, United States
-
Stanford Hospital- Site Number : 8400006
Stanford, California, 94305, United States