Purpose of the Kayak Study
The purpose of the Kayak Study is to evaluate the efficacy, safety, and tolerability of an investigational study drug compared to a placebo (inactive drug) as add-on therapy in young people with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE). The study looks at how the study drug affects things such as your or your child’s symptoms, quality of life, severity of seizures, and frequency of seizures.
Who May Qualify
Eligible participants must meet the following criteria, in addition to other criteria:
- Be male or female 2 to 21 years of age, inclusive.
- Have a diagnosis of SCN8A-DEE supported by both clinical and genetic findings.
- Have at least 1 countable motor seizure (defined as a generalized tonic-clonic seizure [aka grand mal seizure], tonic, atonic or focal [aka partial] onset seizures with noticeable motor component [e.g., involving muscle contractions, a lack of movement, etc.]) per week, on average, and not be seizure-free for more than 20 consecutive days.
- Be receiving treatment with at least 1 other anti-seizure medication (ASM), but no more than 4 ASMs.
- Have failed to achieve seizure freedom with at least 2 previously tried ASMs.
- Not have symptoms that would be more consistent with another epilepsy disorder such as Dravet syndrome (e.g., fever-induced episodes of status epilepticus, frequent myoclonic seizures, worsening on sodium channel blockers, absence seizures with generalized spike-and-wave electroencephalogram [EEG] as the sole seizure type).
- Not be currently taking cannabinoids (other than Epidiolex®/Epidyolex®) or systemic steroids.
Additional eligibility requirements apply. For more information on the study, please click here.
Participants will receive the study drug for up to 18 weeks. In the first 6 weeks, participants will receive increasing doses of the study drug (titration period) based on weight, followed by 10 weeks of treatment at their final tolerated dose (maintenance period) and 2 weeks of treatment with decreasing doses (taper period).
Throughout the study, the study doctor may perform a number of tests and procedures. You can find out more about the study procedures from your study doctor if you or your child is eligible for the study.
Complete the questionnaire