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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

Interested in This Trial?

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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