Status:
RECRUITING
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Lead Sponsor:
Praxis Precision Medicines
Conditions:
Epileptic Encephalopathy
SCN2A Encephalopathy
Eligibility:
All Genders
1-18 years
Phase:
PHASE3
Brief 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 a...
Eligibility Criteria
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.
Key Trial Info
Start Date :
August 13 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT07019922
Start Date
August 13 2025
End Date
December 1 2028
Last Update
October 2 2025
Active Locations (2)
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1
Praxis Research Site
San Diego, California, United States, 92123
2
Praxis Research Site
Chicago, Illinois, United States, 60612