NCT07019922PHASE3Recruiting
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Praxis Precision Medicines
Start Date
8/13/2025
Completion Date
12/1/2028
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
Eligibility Criteria
Age Range: No minimum to 18 years
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.
Interventions
DRUG
1mg elsunersen
DRUG
0.5mg elsunersen
Conditions
Epileptic EncephalopathySCN2A Encephalopathy
Locations
Praxis Research Site
San Diego, California 92123
United States
Praxis Research Site
Aurora, Colorado 80045
United States
Praxis Research Site
Chicago, Illinois 60612
United States
Praxis Research Site
Cincinnati, Ohio 45229
United States
Praxis Research Site
Philadelphia, Pennsylvania 19104
United States
Praxis Research Site
Memphis, Tennessee 38105
United States
Praxis Research Site
Porto Alegre, 90035
Brazil
Praxis Research Site
São Paulo, 05403
Brazil
Praxis Research Site
Bonn, Bonn 53127
Germany
Praxis Research Site
Rome, 00165
Italy