NCT07019922 A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
| NCT ID | NCT07019922 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Praxis Precision Medicines |
| Condition | Epileptic Encephalopathy |
| Study Type | INTERVENTIONAL |
| Enrollment | 40 participants |
| Start Date | 2025-08-13 |
| Primary Completion | 2026-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 40 participants in total. It began in 2025-08-13 with a primary completion date of 2026-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
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.
Contact & Investigator
Medical Director
STUDY DIRECTOR
Praxis Precision Medicines
Frequently Asked Questions
Who can join the NCT07019922 clinical trial?
This trial is open to participants of all sexes, aged 1 Day or older, up to 18 Years, studying Epileptic Encephalopathy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07019922 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 40 participants.
Is NCT07019922 currently recruiting?
Yes, NCT07019922 is actively recruiting participants. Contact the research team at clinicaltrials@praxismedicines.com for enrollment information.
Where is the NCT07019922 trial being conducted?
This trial is being conducted at San Diego, United States, Aurora, United States, Chicago, United States, Cincinnati, United States and 6 additional locations.
Who is sponsoring the NCT07019922 clinical trial?
NCT07019922 is sponsored by Praxis Precision Medicines. The principal investigator is Medical Director at Praxis Precision Medicines. The trial plans to enroll 40 participants.