A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Voltage-gated sodium channel type II alpha subunit (SCN2A) developmental and epileptic encephalopathy (DEE)

Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks

Responder rate - defined as a ≥50% reduction in monthly seizure frequency from baseline compared to treatment after 24 weeks, Change in motor seizure-free days from baseline (Treatment and Extension Periods), Clinical Global Impression-Severity (CGI-S) at baseline compared to treatment after 24 weeks (Treatment and Extension Periods), Clinical Global Impression-Improvement (CGI-I) subdomains scores at each postdose time point (Treatment and Extension Periods), Caregiver Global Impression-Severity (CgGI-S) at baseline compared to treatment after 24 weeks (Treatment and Extension Periods), Caregiver Global Impression-Improvement (CgGI-I) subdomains scores at each postdose time point (Treatment and Extension Periods), Sleep assessment scores at baseline compared to each postdose time point (Treatment and Extension Periods), Incidence and severity of treatment-emergent adverse events (TEAEs) (Treatment and Extension Periods), Changes in findings on physical and neurological examinations, vital sign measurements, clinical laboratory results and electrocardiogram (ECG) parameters (Treatment and Extension Periods), Plasma and cerebrospinal fluid (CSF) concentrations of elsunersen, Plasma elsunersen PK parameters, Changes in the ASM regimen, dose and number of ASMs compared to Baseline (Extension Period Only)

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Germany, Italy