Gene therapy study for Charcot-Marie-Tooth type 4J (CMT4J)
Part of Brain & nervous system clinical trials.
This trial tests a gene therapy medicine (ELP-02) given through a spinal tap for children and young adults with a specific genetic form of CMT (CMT4J). It aims to slow or stop the disease from getting worse.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 3 to 20 years old
- Must have a confirmed genetic diagnosis of CMT4J with one specific mutation (I41T) in the FIG4 gene
- Must have symptoms of motor weakness or other CMT4J signs
- Disease should not be too severe (CMTPeds score less than 35 or doctor's judgment)
- Must be willing to undergo a lumbar puncture (spinal tap) and use birth control if sexually active
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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