Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07019922 | A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety | PHASE3 | RECRUITING | 40 | — | — | Aug 13, 2025 | Dec 1, 2028 | May 27, 2026 | 10 | United States, Brazil +2 |
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
| Arm | Type | Description |
|---|---|---|
| Cohort 1 Arm 1: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 2: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 3: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Name | Type | Description |
|---|---|---|
| 1mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| 0.5mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
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 cli...