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.
The Kayak Study is currently on a pre-planned enrollment pause to review the safety and tolerability data of the first 8 enrolled participants. Based on this data, we are in the process of implementing changes to the overall trial. Once a date for re-opening enrollment has been set, we will post it on the website.
Please complete the brief online questionnaire if you would like to register your interest in the trial once enrollment re-opens. This questionnaire will help determine if you or your child may qualify for the trial. If the questionnaire indicates you or your child may qualify, you will be connected with a trial site once enrollment re-opens.
Please note that you may not hear from the trial site until the re-opening of enrollment.
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.