NCT07019922

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

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Praxis Precision Medicines · Industry
• Sex: All
• Age: 1 Day – 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Epileptic Encephalopathy
• SCN2A Encephalopathy

Interventions

Type	Name	Description
DRUG	1mg elsunersen	24 weeks every 4 weeks intrathecally
PROCEDURE	sham procedure	24 weeks of sham-procedure every 4 weeks
DRUG	0.5mg elsunersen	24 weeks every 4 weeks intrathecally

Timeline

• Start date: 2025-08-13
• Primary completion: 2026-06-01
• Completion: 2028-12-01
• First posted: 2025-06-13
• Last updated: 2025-10-02

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study