Clinical Trials and Emerging Treatments
Neurocrine Biosciences has the first drug in clinical trials for SCN8A developmental and epileptic encephalopathy (SCN8A-DEE). This drug is a specifically designed to treat those with SCN8A-DEE and clinical trial research is underway (via the Kayak Study) to test how effective it is for those living with the disorder.
The Kayak Study is now underway
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 2 to 21 years of age with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE). The study is looking at how the study drug affects participants’ symptoms, quality of life, severity of seizures, and frequency of seizures. To see if you or your child may qualify for study participation, click here.
If your healthcare provider is interested in learning more about this study, they should contact the Neurocrine Medical Information Center at firstname.lastname@example.org or 1-877-641-3461.
The Kayak Study is recruiting in 2 waves. The first group is called the sentinel cohort. These first 8 people are being recruited and enrolled in the study via sites in the United States. Data from these first 8 people will be analyzed by an independent data review committee and once they are satisfied of the safety and tolerability of the study drug and any necessary adjustments are made, recruitment will begin for up to another 52 people with SCN8A-DEE. Recruitment of this main cohort will likely begin in early 2023 on a global level at sites throughout the U.S., Australia, Belgium, Canada, Czech Republic, Denmark, France, Germany, Italy, Netherlands, Poland, Portugal, Spain and the United Kingdom.