ION337 gene therapy for children with Dravet syndrome
Part of Brain & nervous system clinical trials.
This study tests a new gene therapy called ION337 designed to treat Dravet syndrome, a severe childhood epilepsy caused by a SCN1A gene mutation. The therapy is given as a spinal fluid injection and aims to reduce seizures in children ages 2–12.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is between 2 and 12 years old
- Your child has been diagnosed with Dravet syndrome confirmed by genetic testing (SCN1A gene mutation)
- Your child is currently taking at least one anti-seizure medication at a stable dose for at least 4 weeks
- All other epilepsy treatments (like special diet or nerve stimulator) and other medications have been stable for at least 4 weeks
- Your child has had a certain number of major seizures during the screening period
- You have not enrolled in another gene therapy or antisense oligonucleotide trial for Dravet syndrome
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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