Clinical Trials and Emerging Treatments
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. The study is now paused but to see if you or your child may qualify for future study participation, click here.
The Kayak Study is recruiting in 2 waves. The first group is called the sentinel cohort, which has already been recruited. These first 8 people were recruited and enrolled in the study via sites in the United States. Data from these first 8 people is now being analyzed by an independent data review committee and once they are satisfied with 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 mid-late 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.
Praxis Precision Medicine
Recruitment Closed – The PACIFIC study
The PACIFIC Study is a clinical trial evaluating participants with DEE. The primary objectives of the study are to assess the safety & tolerability of an investigational study drug. The PACIFIC study is also designed to examine change in seizure frequency over the 90-day treatment period. The study plans to enroll approximately 50 participants with a variety of treatment resistant seizures that fall into the category of DEEs. Approximately 35 study sites in the United States and Australia will participate.