Clinical Trials and Emerging Treatments

Neurocrine Biosciences
The purpose of the Kayak Study is to evaluate the efficacy, safety
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 participantsโ 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,ย click here.ย
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 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
In January 2022,ย Praxis published aย paper with pre-clinical data on PRAX-562, a persistent current sodium channel blocker that is in development to treat a range of developmental and epileptic encephalopathies (DEEs), including SCN8A-DEE.
The EMBOLD Study is now pre-screening for participation in this study.