Study of Nexviazyme in children with infantile Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be under 18 years old.
- Pompe symptoms started before 12 months of age.
- Heart muscle problems (cardiomyopathy) were present at diagnosis.
- Your child can have received either Myozyme/Lumizyme or Nexviazyme before, but not other Pompe treatments.
- If your child has had a different enzyme therapy (not Myozyme, Lumizyme, or Nexviazyme), they cannot join.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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