Testing a new spinal injection treatment for severe childhood seizures

This trial is testing a new medication called ION337 that is given as an injection into the spinal fluid to help reduce seizures in children with Dravet syndrome, a rare and severe form of epilepsy. The study will first give one dose to small groups of children at different dose levels to see if the treatment is safe, and then move to giving multiple doses over a longer period. Researchers want to understand how the medication works in the body and whether it can safely help children have fewer seizures.

Dravet syndrome is a severe, hard-to-treat form of childhood epilepsy where seizures are difficult to control with current medications. This trial is testing whether this new treatment, delivered directly into the spinal fluid, can help children with this condition have fewer seizures and live safer, healthier lives.