Less frequent infusions for Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests if getting enzyme replacement therapy every 4 weeks instead of every 2 weeks is safe and effective for stable adults with late-onset Pompe disease. It may reduce the burden of frequent infusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have late-onset Pompe disease, confirmed by a lab test or genetic test
- You are 50 years or older
- You have been on alglucosidase alfa (Myozyme) infusions every 2 weeks for at least 4 years
- Your condition has been mostly stable over the last year
- You can walk at least 150 meters (about 1.5 football fields) in 6 minutes
- Your lung function is at least 45% of expected when lying down and 55% when sitting
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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