A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Key facts
- Where
- Rome, ItalySee the site
- Phase
- Phase 3
Where in Italy
- RomeRome, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Praxis Precision Medicines
- Trial code
- NCT07019922
- Record updated
- 3 August 2026
- Original description
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 EncephalopathyRead full original descriptionShow less
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
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
Inclusion: 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: 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; 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; Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.