NCT07019922RecruitingPhase 3
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
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
Source: ClinicalTrials.gov · Last updated 3 Aug 2026
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 5 Jun 2025
- Start date
- 13 Aug 2025
- Primary completion date
- 31 Dec 2026
- Lead sponsor
- Praxis Precision Medicines
- Diseases
- Epilepsy
- NCT number
- NCT07019922
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 40
- From 13 Aug 2025 to the primary completion date, 31 Dec 2026
- 10 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- 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.
Other
- Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.
Who the study is looking for
- 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.
Age in the registry: 1 day to 18 years · Sex: All · Does not accept healthy volunteers
Sites (10)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Praxis Research Site Coordinator of this site? Confirm its status | Porto Alegre | Brazil | Withdrawn |
| Praxis Research Site Coordinator of this site? Confirm its status | São Paulo | Brazil | Withdrawn |
| Praxis Research Site Coordinator of this site? Confirm its status | Bonn | Germany | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Rome | Italy | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Aurora | United States | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Chicago | United States | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Cincinnati | United States | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Memphis | United States | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| Praxis Research Site Coordinator of this site? Confirm its status | San Diego | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.