Skip to content
Bored Studies
RecruitingPhase 3

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

Praxis Precision Medicines · NCT07019922

View Study & Apply (opens in a new tab)

Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 0 mo–18 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
40
US sites recruiting
6
Study start
August 13, 2025
Record last updated
August 3, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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

Being studied: 1mg elsunersen; 0.5mg elsunersen

Conditions: Epileptic Encephalopathy; SCN2A Encephalopathy

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

Study contacts (from the official record)

  • Head of Pharmacovigilance · 617-300-8460 · clinicaltrials@praxismedicines.com
All neurology studies in California →
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess… (NCT07019922) | Bored Studies