Back to search results

Elsunersen for Pediatric SCN2A-DEE

This study is testing a drug called elsunersen in children and teenagers (ages 1 day to 18 years old) who have a specific type of epilepsy called SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE). This condition is caused by a change in the SCN2A gene and leads to seizures starting before 3 months of age. The study wants to see how well elsunersen works to reduce the number of seizures and if it is safe. To join, participants must have a confirmed SCN2A gene change, seizures that started before 3 months old, and at least four seizures in a 28-day period. The study is currently unclear on its recruitment status and plans to enroll 40 participants.

Study design
This is a single-arm study, meaning all participants will receive elsunersen. It plans to enroll 40 participants.
What's involved
Participants will receive elsunersen injections into their spinal fluid every 4 weeks for 24 weeks.
Compensation
Not stated in the trial record.
Follow-up
The main goal of the study is measured at 24 weeks after starting treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07019922

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

Recruiting
PHASE3Ages 1–18InterventionalTreatment
Praxis Precision Medicines
~40 participants
Updated 2026-08-03 on ClinicalTrials.gov
What's tested:1mg elsunersen0.5mg elsunersen

At a glance

Recruiting sites
8 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
Measured over 24 weeks

Conditions

Epileptic Encephalopathy
SCN2A Encephalopathy

Explore related trials

SYNGAP1-related developmental and epileptic encephalopathySCN2A Gene

NCT07019922

Where you'd take part

This study runs at 10 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Praxis Research Site

    San Diego, Californiano site contact published

    Recruiting

  • Praxis Research Site

    Aurora, Coloradono site contact published

    Recruiting

  • Praxis Research Site

    Chicago, Illinoisno site contact published

    Recruiting

  • Praxis Research Site

    Cincinnati, Ohiono site contact published

    Recruiting

  • Praxis Research Site

    Philadelphia, Pennsylvaniano site contact published

    Recruiting

  • Praxis Research Site

    Memphis, Tennesseeno site contact published

    Recruiting

  • Praxis Research Site

    Bonn, Bonn, Germanyno site contact published

    Recruiting

  • Praxis Research Site

    Rome, Italyno site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Medical Director · STUDY_DIRECTOR · Praxis Precision Medicines

Who to contact

Head of Pharmacovigilance
[email protected]
617-300-8460
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Want this trial checked against your situation?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

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

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.

What this trial measures

  • To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE24 weeks

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