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

Phase 3

Not yet recruiting

• Conditions: SCN2A Encephalopathy; Epileptic Encephalopathy
• Interventions: Drug: 1mg elsunersen; Procedure: sham procedure; Drug: 0.5mg elsunersen

• Registration Number: NCT07019922
• Lead Sponsor: Praxis Precision Medicines

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-06
• Study Start: 2025-06
• Study First Submitted: 2025-06-05
• Study First Submitted QC: 2025-06-05
• Study First Posted: 2025-06-13
• Last Update Submitted: 2025-06-13
• Last Update Posted: 2025-06-18
• Primary Completion: 2026-06
• Study Completion: 2028-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Has a documented Gain of Function SCN2A variant confirmed through genetic testing.
• Has onset of seizures prior to 3 months of age.
• Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period.

Exclusion Criteria

• Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
• Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder.
• Has received any experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
• Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1 Arm 1: Double-Blind Treatment Period	1mg elsunersen	Double-blind treatment period elsunersen
Cohort 1 Arm 2: Double-Blind Treatment Period	sham procedure	Double-blind sham-procedure
Cohort 2: Open-Label Treatment Period	1mg elsunersen	Open-label elsunersen
Cohort 3: Open-Label Treatment Period	0.5mg elsunersen	Open-label elsunersen

Primary Outcome Measures

Name	Time	Method
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE	24 weeks	Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks

Secondary Outcome Measures

Name	Time	Method
CgGI-I subdomain scores at each postdose time point	24 weeks	Caregiver Global Impression-Improvement (CgGI I) subdomains scores at each postdose time point
To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE	24 weeks	Change in motor seizure-free days from baseline
CGI-S change from baseline	24 weeks	CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this patient population and rate the current severity on a 7-point scale from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).
CGI subdomain scores at each postdose time point	24 weeks	Clinical Global Impression-Improvement (CGI-I) subdomains scores at each postdose time point
CgGI-S from baseline	24 weeks	Caregiver Global Impression-Severity (CgGI-S) at baseline compared to treatment after 24 weeks
To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE	24 weeks	Incidence and severity of treatment-emergent adverse events (TEAEs)
Sleep assessment scores from baseline	24 weeks	Sleep assessment scores at baseline compared to each postdose time point

Trial Locations

Locations (1)

Praxis Research Site; 🇺🇸 Chicago, Illinois, United States