In short
Clinical trials are investigating "5'-MOEMC-(SP)-MOEMC-(P)-MOEA-(P)-MOEMC-(P)-MOEG-(P)-MOEA-(P)-DMC-(SP)-DA-(SP)-DT-(SP)-DA-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DMC-(SP)-MOET-(P)-MOEA-(SP)-MOEMC-(SP)-MOEA 3'" in children with early-onset SCN2A developmental and epileptic encephalopathy (DEE). These studies aim to evaluate efficacy, safety, and pharmacokinetics, which means how the treatment works, how safe it is, and how the body handles it.
Key points
- Clinical trials are investigating "5'-MOEMC-(SP)-MOEMC-(P)-MOEA-(P)-MOEMC-(P)-MOEG-(P)-MOEA-(P)-DMC-(SP)-DA-(SP)-DT-(SP)-DA-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DT-(SP)-DMC-(SP)-MOET-(P)-MOEA-(SP)-MOEMC-(SP)-MOEA 3'" in children with early-onset SCN2A developmental and epileptic encephalopathy. The available trial is a Phase 3 interventional study with 40 participants. It is designed to assess efficacy, safety, and pharmacokinetics of elsunersen. The main outcome is the change in monthly motor seizure frequency after 24 weeks compared with the starting point. The trial status is authorised.
Trial overview
This study is an interventional trial, which means researchers are giving a treatment and then measuring the results. The trial title says it is looking at the efficacy, safety, and pharmacokinetics of elsunersen in children.
The condition being studied is voltage-gated sodium channel type II alpha subunit (SCN2A) developmental and epileptic encephalopathy, also called SCN2A-DEE. The brief summary says the study is focused on participants with early-onset SCN2A-DEE.
Who can participate
The trial is for children. It targets people with early-onset SCN2A-DEE, so the main group is children whose seizure disorder began early in life.
- Target population: children with early-onset SCN2A-DEE.
- Study size: 40 participants are planned.
What is being measured
The main endpoint is the change in monthly motor seizure frequency from baseline to after 24 weeks. In simple words, the study checks whether the number of motor seizures changes after treatment compared with the starting point.
Motor seizure frequency means how often seizures with body movement happen over a 28-day period. Baseline means the first measurement before treatment starts.
Trial status and phase
The trial status is Authorised. It is a Phase 3 study, which usually means the treatment is being tested in a larger group to learn more about how well it works and to keep collecting safety information.
The study is listed as a pharmacokinetics trial as well, so it also looks at how the body handles the treatment.
Key patient terms
- SCN2A-DEE: a rare brain disorder linked to the SCN2A gene that causes seizures and developmental problems.
- Efficacy: how well a treatment works.
- Safety: how well a treatment can be used without unacceptable harm.
- Pharmacokinetics: how the body absorbs, moves, and removes a treatment.
- Interventional trial: a study where a treatment is given to see what happens.