Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
STK-001- Single Ascending Doses · 1 trial · 1 indication
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 *...
STK-001 - Single Ascending Doses is an investigational therapy being studied for the treatment of Dravet syndrome, a severe form of epilepsy that begins in infancy. It is being developed by Stoke Therapeutics, Inc. and is in Phase 1 clinical development. The single ascending doses portion refers to a study design that evaluates the safety of increasing dose levels.
STK-001 - Single Ascending Doses is being developed by Stoke Therapeutics, Inc., which trades on the Nasdaq under the ticker symbol STOK. The company is focused on developing novel RNA-based medicines. The drug candidate is in Phase 1 clinical development for Dravet syndrome.
STK-001 - Single Ascending Doses is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 1 study evaluating single and multiple ascending doses in children and adolescents with Dravet syndrome has been completed.
STK-001 - Single Ascending Doses has been evaluated in one completed Phase 1 clinical trial, NCT04442295. The trial was an open-label study investigating the safety of single and multiple ascending doses in children and adolescents with Dravet syndrome. It enrolled 62 participants in the United States and has been completed.
STK-001 - Single Ascending Doses is also known as STK-001 - Single Ascending Doses. The name refers to the specific Phase 1 study design evaluating single ascending doses of the investigational therapy. It is being developed by Stoke Therapeutics for Dravet syndrome.