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

A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Who can join

1 Day – 18 Years · All sexes

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.

About the study

A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

What is being tested

• 1mg elsunersen (drug)
• 0.5mg elsunersen (drug)

Sponsor: Praxis Precision Medicines · Participants: 40 · Started: Aug 13, 2025

Contact the study team

• Head of Pharmacovigilance · Phone: 617-300-8460

Official record on ClinicalTrials.gov — NCT07019922

Locations in the U.S.

Conditions

• Epilepsy
• Seizures

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.