Actively Recruiting
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Led by Praxis Precision Medicines · Updated on 2026-05-13
50
Participants Needed
4
Research Sites
172 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Praxis Research Site
San Diego, California, United States, 92123
Actively Recruiting
2
Praxis Research Site
Chicago, Illinois, United States, 60612
Actively Recruiting
3
Praxis Research Site
Porto Alegre, Brazil, 90035
Actively Recruiting
4
Praxis Research Site
São Paulo, Brazil, 05403
Actively Recruiting
Research Team
H
Head of Pharmacovigilance
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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