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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04442295 | An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome | PHASE1 | COMPLETED | 62 | — | — | Jun 29, 2020 | Nov 14, 2023 | May 18, 2025 | 18 | United States |
1. Incidence of adverse events 2. incidence of abnormal vital signs 3. Abnormal physical examination findings 4. Abnormal 12-lead electrocardiogram (ECG) 5. Abnormal laboratory parameters
Analysis of plasma concentrations of STK-001
Measurement of STK-001 concentrations
| Arm | Type | Description |
|---|---|---|
| Single Ascending Doses | EXPERIMENTAL | Enrollment of patients in two age groups. A Sentinel group of 2 patients aged 13 to 18 years of age, inclusive, and an expanded group of 2 patients 2 to 12 years of age to receive single doses. There will be an option to dose up to 6 additional patients at each dose level and an option to expand the maximum tolerated dose level with 5 additional patients. |
| Multiple Ascending Doses | EXPERIMENTAL | Enrollment of patients in two age groups. A Sentinel group of 2 patients aged 13 to 18 years of age, inclusive, and an expanded group of 2 patients 2 to 12 years of age to receive multiple doses. There will be an option to dose up to 6 additional patients at each dose level and an option to expand the maximum tolerated dose level with 10 additional patients. |
| Name | Type | Description |
|---|---|---|
| STK-001 - Single Ascending Doses | DRUG | Experimental : Single Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Four dose levels will be evaluated ( 10mg, 20mg,30mg, 45mg and 70mg ). |
| STK-001 - Multiple Ascending Doses | DRUG | Experimental : Multiple Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Three dose levels will be evaluated ( 20mg,30mg and 45mg ). |
Inclusion Criteria: * Diagnosis of Dravet Syndrome (DS) with onset of recurrent focal motor or hemiconvulsive or generalized tonic-clonic seizures prior to 12 months of age, which are often prolonged and triggered by hyperthermia. * No history of causal MRI lesion * No other known etiology *...