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
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
- Age range
- 0–18 years
- Sex
- All
- Healthy volunteers
- No
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 experime…
Interventions
- Drug1mg elsunersen
24 weeks every 4 weeks intrathecally
- Proceduresham procedure
24 weeks of sham-procedure every 4 weeks
- Drug0.5mg elsunersen
24 weeks every 4 weeks intrathecally
Locations (2)
- Praxis Research SiteSan Diego, California
- Praxis Research SiteChicago, Illinois