New hope for babies with rare muscle disease: enzyme therapy trial launches in china
NCT ID NCT06666413
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and heart problems. Participants will receive the drug through an IV every two weeks for about a year. The goal is to see if the treatment is safe and helps children survive without needing a breathing machine.
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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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Study contacts
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Contact
Email: •••••@•••••
Locations
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Investigational Site Number: 1560001
RECRUITINGShanghai, 200127, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New enzyme therapy gives hope to babies with rare muscle disease
- 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