MedTrace Logo

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

NCT07019922 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-05-13

No results posted yet for this study

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

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

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Praxis Precision Medicines

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
1 Day
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-13
Primary Completion
2026-06-30
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States
  • Brazil

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07019922 on ClinicalTrials.gov