Elsunersen treatment for infants with early-onset SCN2A-related epileptic encephalopathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, multi-center, double-blind, sham-procedure-controlled Phase 3 trial comparing two doses of elsunersen (1 mg and 0.5 mg) versus a sham procedure in pediatric participants with early-onset SCN2A-DEE. Eligible participants must have a confirmed gain-of-function SCN2A variant, seizure onset before 3 months of age, and at least 4 countable motor seizures per 28 days during baseline. Participants are randomized to one of the intervention arms and followed with regular visits to track seizure counts and monitor safety and adverse events. The trial will compare changes in seizure frequency and safety signals between the elsunersen dose groups and the sham control over the treatment period.

Why it matters

Who can join: the full rules

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.

Where it's running

• Praxis Research Site
  San Diego, California, United States Recruiting
• Praxis Research Site
  Aurora, Colorado, United States Recruiting
• Praxis Research Site
  Chicago, Illinois, United States Recruiting
• Praxis Research Site
  Cincinnati, Ohio, United States Recruiting
• Praxis Research Site
  Philadelphia, Pennsylvania, United States Recruiting
• Praxis Research Site
  Memphis, Tennessee, United States Recruiting

Study contacts

• Study coordinator: Head of Pharmacovigilance
• Email: clinicaltrials@praxismedicines.com
• Phone: 617-300-8460

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.