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A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Sponsor: Praxis Precision Medicines
Summary
A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy
Official title: A Multi-Center, Single-Arm 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
40
Start Date
2025-08-13
Completion Date
2028-12
Last Updated
2026-05-27
Healthy Volunteers
No
Interventions
1mg elsunersen
24 weeks every 4 weeks intrathecally
0.5mg elsunersen
24 weeks every 4 weeks intrathecally
Locations (10)
Praxis Research Site
San Diego, California, United States
Praxis Research Site
Aurora, Colorado, United States
Praxis Research Site
Chicago, Illinois, United States
Praxis Research Site
Cincinnati, Ohio, United States
Praxis Research Site
Philadelphia, Pennsylvania, United States
Praxis Research Site
Memphis, Tennessee, United States
Praxis Research Site
Porto Alegre, Brazil
Praxis Research Site
São Paulo, Brazil
Praxis Research Site
Bonn, Bonn, Germany
Praxis Research Site
Rome, Italy