Testing a new drug for Lafora disease in young people
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental drug called ION283, given by a lumbar puncture (spinal tap), to see if it is safe and can help with Lafora disease symptoms. It is for patients aged 10 to 18 with a confirmed genetic diagnosis.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 10 to 18 years old and have a confirmed genetic diagnosis of Lafora disease.
- You or your parent/guardian must agree to the study rules and sign a consent form.
- You must be able to walk at least 10 steps on your own.
- You cannot have certain medical conditions like bleeding problems, active infections, or severe kidney or liver issues.
- You must not have used certain blood-thinning medications or other experimental treatments within a specific time before the study.
- Women who could become pregnant must use highly effective birth control during the study and for 12 weeks after the last dose.
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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