A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

Recruiting now · Phase 3

Conditions studied: Epileptic Encephalopathy, SCN2A Encephalopathy

In brief

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 facts

Study ID

NCT07019922

Run by

Praxis Precision Medicines

People needed

40

Starts

2025-08-13

Expected to finish

2028-12-01

Last updated by the study team

2026-08-03

Who can join

Age: 0 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify if…

• 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.

Where it is running

• Praxis Research Site — Rome, Italy (enrolling)
• Praxis Research Site — Aurora, Colorado, United States (enrolling)
• Praxis Research Site — Chicago, Illinois, United States (enrolling)
• Praxis Research Site — Cincinnati, Ohio, United States (enrolling)
• Praxis Research Site — Philadelphia, Pennsylvania, United States (enrolling)
• Praxis Research Site — Memphis, Tennessee, United States (enrolling)
• Praxis Research Site — Bonn, Bonn, Germany (enrolling)
• Praxis Research Site — San Diego, California, United States (enrolling)
• Praxis Research Site — Porto Alegre, Brazil
• Praxis Research Site — São Paulo, Brazil

Full record on ClinicalTrials.gov

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