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 is an investigational small molecule being studied for the treatment of Dravet Syndrome, a severe form of childhood epilepsy. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is designed to address the underlying genetic cause of the condition, though its exact mechanism is not specified.
STK-001 is being developed by Stoke Therapeutics, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol STOK. The company is conducting clinical trials to evaluate the safety and tolerability of STK-001 in pediatric patients with Dravet Syndrome.
STK-001 is currently in Phase 1 clinical development. A Phase 1 study has been completed, which evaluated the safety of single and multiple ascending doses in children and adolescents with Dravet Syndrome. The drug remains investigational and has not received FDA approval.
STK-001 has one completed clinical trial registered under NCT04442295, titled "An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome." This Phase 1 study enrolled 62 participants in the United States and included children as young as 2 years old.
Yes, STK-001 is the same drug as STK-001- Single Ascending Doses. The latter name refers to the specific dosing regimen evaluated in the clinical trial. The drug is being developed by Stoke Therapeutics for the treatment of Dravet Syndrome.