A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

To join this study, children and young people need to meet certain requirements. First, a genetic test must have confirmed that they have a specific type of change in the SCN2A gene that is known to cause a particular problem with how the gene works. Also, their seizures must have started before they were three months old. Finally, they must be experiencing at least four or more noticeable seizures (where a motor action, like jerking, happens) every 28 days during a special observation period before the study officially begins.

There are also reasons why someone might not be able to join. For example, if they have other significant genetic conditions besides the SCN2A gene change that could be causing their epilepsy or developmental problems. Children with other serious health issues, like problems with their bones, spine (like curves in the back), or difficulties with bleeding, would also not be able to take part.

Additionally, if a child has recently received another experimental or unproven treatment or device, or if they are pregnant or breastfeeding, or planning to become pregnant, they would not be eligible for this study.

If you decide to take part, your child will receive one of two doses of the study medicine, elsunersen (either 1mg or 0.5mg). They will be closely monitored throughout the study. This will involve regular visits to the study clinic where doctors and nurses will perform assessments such as physical examinations, neurological checks, and blood tests. They will also keep track of your child's seizures.

You or your child's caregiver will be asked to keep a detailed diary of all seizures your child experiences. The total duration of participation in the study will be explained to you by the study doctor, including how long the treatment period lasts and any follow-up visits. This information will be fully discussed during your initial meetings with the study team.