Table of contents
Trial overview
This study is an interventional trial, which means researchers are giving a treatment and then measuring the results.[1] The trial title says it is looking at the efficacy, safety, and pharmacokinetics of elsunersen in children.[1]
The condition being studied is voltage-gated sodium channel type II alpha subunit (SCN2A) developmental and epileptic encephalopathy, also called SCN2A-DEE.[1] The brief summary says the study is focused on participants with early-onset SCN2A-DEE.[1]
Who can participate
The trial is for children.[1] It targets people with early-onset SCN2A-DEE, so the main group is children whose seizure disorder began early in life.[1]
- Target population: children with early-onset SCN2A-DEE.[1]
- Study size: 40 participants are planned.[1]
What is being measured
The main endpoint is the change in monthly motor seizure frequency from baseline to after 24 weeks.[1] In simple words, the study checks whether the number of motor seizures changes after treatment compared with the starting point.[1]
Motor seizure frequency means how often seizures with body movement happen over a 28-day period.[1] Baseline means the first measurement before treatment starts.[1]
Trial status and phase
The trial status is Authorised.[1] 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.[1]
The study is listed as a pharmacokinetics trial as well, so it also looks at how the body handles the treatment.[1]
Key patient terms
- SCN2A-DEE: a rare brain disorder linked to the SCN2A gene that causes seizures and developmental problems.[1]
- Efficacy: how well a treatment works.[1]
- Safety: how well a treatment can be used without unacceptable harm.[1]
- Pharmacokinetics: how the body absorbs, moves, and removes a treatment.[1]
- Interventional trial: a study where a treatment is given to see what happens.[1]
