What is elsunersen studying for early-onset SCN2A Developmental and Epileptic Encephalopathy?

Elsunersen is being studied to evaluate its effects on seizure frequency and other clinical outcomes in children with early-onset SCN2A Developmental and Epileptic Encephalopathy. The trial aims to understand how this treatment may impact seizures and related symptoms in this pediatric condition. Praxis Precision Medicines is sponsoring this research.

Who may be eligible for this SCN2A encephalopathy clinical trial?

Children from birth to 18 years old with a confirmed gain of function SCN2A genetic variant and seizures starting before 3 months of age may be eligible. Participants must have at least 4 motor seizures per month and should not be pregnant or breastfeeding. Please review eligibility criteria for more details.

Is there any cost to participate in this SCN2A encephalopathy clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 3

Age: 1Day - 18Years

All Genders

ID07019922

Study of Elsunersen Treatment in Children With Early-Onset SCN2A Developmental and Epileptic Encephalopathy

Led by Praxis Precision Medicines · Updated on 2026-08-03

Participants Needed

Research Sites

104 weeks

Total Duration

AI-Summary

What this Trial Is About

This clinical trial evaluates the efficacy and safety of elsunersen in children with early-onset SCN2A Developmental and Epileptic Encephalopathy, a condition characterized by seizures beginning before 3 months of age. The study focuses on pediatric participants confirmed to have a gain of function SCN2A genetic variant, aiming to understand how elsunersen impacts seizure frequency and related symptoms. The trial is sponsored by Praxis Precision Medicines and is conducted at multiple centers. Participants receive intrathecal doses of elsunersen every 4 weeks for a total of 24 weeks. Two dosing levels, 1mg and 0.5mg, are being studied in an open-label design across three cohorts. All participants receive the study drug without a placebo comparison, and treatment effects on seizures and other clinical assessments are monitored throughout the treatment period. During the 24-week treatment, participants undergo regular evaluations to assess seizure frequency, clinical global impression of severity and improvement, and sleep quality among other outcomes. Safety and tolerability are also closely monitored. The trial includes baseline observation periods and follow-up assessments to track changes over time, providing a comprehensive view of elsunersens impact in this pediatric population.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 1Day - 18Years

All Genders

Eligibility Criteria

You may qualify if you...

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.

You will not qualify if you...

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.

Research Team

Head of Pharmacovigilance

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here