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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06872125 | A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome | PHASE3 | RECRUITING | 170 | — | — | Jun 4, 2025 | Oct 1, 2028 | Jun 2, 2026 | 61 | United States, France +5 |
| NCT04740476 | An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome | PHASE2 | ACTIVE NOT_RECRUITING | 60 | — | — | Jan 20, 2021 | Mar 31, 2029 | Feb 4, 2026 | 17 | United States |
Measured by daily seizure diary
Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and outcomes on the cerebellar function clinical screening battery.
| Arm | Type | Description |
|---|---|---|
| Zorevunersen | EXPERIMENTAL | Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham in Treatment Period 1 (approximately 52 weeks). Upon the completion of Treatment Period 1 all eligible patients, will enter Treatment Period 2 and receive zorevunersen, regardless of initial treatment assignment. |
| Sham Comparator | SHAM_COMPARATOR | Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham |
| zorevunersen (STK-001) multiple dose levels | EXPERIMENTAL | Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. Patients will receive IT administration of study drug zorevunersen at the dose level they received while participating in Study STK-001-DS-101 or STK-001-DS-102, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an zorevunersen Phase 1/2 study, and doses above 45 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56. |
| Name | Type | Description |
|---|---|---|
| zorevunersen | DRUG | Treatment Period 1: Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281. Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84). |
| Sham Comparator | OTHER | Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration. Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84). |
| zorevunersen (STK-001) | DRUG | zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection. |
Key Inclusion Criteria: 1. Patients must be ≥2 and \<18 years of age. 2. Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months (inclusive, \<13 months), of age, of recurrent focal with motor signs, hemi...