A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
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
Praxis Precision Medicines
50 participants
Aug 13, 2025
INTERVENTIONAL
Conditions
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
Eligibility
Inclusion Criteria3
- 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 Criteria4
- 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.
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Interventions
24 weeks every 4 weeks intrathecally
24 weeks of sham-procedure every 4 weeks
24 weeks every 4 weeks intrathecally
Locations(2)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07019922