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A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Sponsor: 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
Official title: 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
Key Details
Gender
All
Age Range
1 Day - 18 Years
Study Type
INTERVENTIONAL
Enrollment
50
Start Date
2025-08-13
Completion Date
2028-12
Last Updated
2025-10-02
Healthy Volunteers
No
Interventions
1mg elsunersen
24 weeks every 4 weeks intrathecally
sham procedure
24 weeks of sham-procedure every 4 weeks
0.5mg elsunersen
24 weeks every 4 weeks intrathecally
Locations (2)
Praxis Research Site
San Diego, California, United States
Praxis Research Site
Chicago, Illinois, United States