Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency, overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.
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Safety and Tolerability of single and multiple doses of STK-001 with respect to:
Timeframe: Screening (Day -28) until 6 months after single and multiple drug dosing
Pharmacokinetic (PK) Parameters
Timeframe: Day 1 (Dosing) until 6 months after single and multiple drug dosing
Exposure of STK-001 in Cerebrospinal Fluid (CSF)
Timeframe: Day 1 (Dosing) until 6 months after single and multiple drug dosing