Study of DNL952 for adults with late-onset Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new drug, DNL952, in adults with late-onset Pompe disease. It aims to see if the drug is safe and how it works in the body, with different groups for people currently on enzyme replacement therapy or those who haven't had it in the past year.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must weigh at least 40 kilograms (about 88 pounds).
- You have been diagnosed with late-onset Pompe disease.
- Your lung function (measured by FVC) is at least 30% of what is expected for someone your age and height.
- You can walk at least 40 meters (about 130 feet), with or without a walker or cane.
- If you are in Group A, you must have been on one of two standard enzyme replacement therapies (every two weeks) for at least the last year.
- If you are in Group B, you must not have had any enzyme replacement therapy for Pompe disease in the last year.
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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