Clinical Trials & Emerging Treatments

Praxis Precision Medicine

In January 2022, Praxis published a 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 enrolling participants! 

Neurocrine Biosciences

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 (SCN8A-DEE). The trial is evaluating how the study drug affects participants’ symptoms, quality of life, severity of seizures, and frequency of seizures. 

Recruitment of the next cohort will rollout 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.

Longboard Pharmaceuticals

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.

How Do Clinical Trials Work?