Study of ION440 for MECP2 Duplication Syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental medicine called ION440 for people with MECP2 Duplication Syndrome. The medicine is given as an injection into the spinal fluid, and the study aims to check its safety and how well it works.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a male between 2 and 65 years old.
- You need a confirmed genetic diagnosis of MECP2 Duplication Syndrome.
- Your current medications must be stable for at least one month.
- You should not have severe disease features like oxygen dependence or seizures before age 5.
- You cannot have had gene therapy or cell transplantation in the past.
- You must have no other medical conditions that would make the lumbar puncture unsafe.
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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