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 medinfo@neurocrine.com 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.

Sites Currently Recruiting Include:

UCSF Medical Center Recruiting
San Francisco, California, United States, 94158
Contact: Lucy Liu    415-353-2437    Lucy.liu3@ucsf.edu   

Children’s National Hospital Recruiting
Washington, District of Columbia, United States, 20010
Contact: Zachary Kramer       zkramer@childrensnational.org   

Ann & Robert H. Lurie Children’s Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Savannah O’Neil       sroneil@luriechildrens.org   

University of Rochester Recruiting
Rochester, New York, United States, 14642
Contact: Noreen Connolly       Noreen_Connolly@urmc.rochester.edu   
Children’s Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jennie Minnick       ENGIN@chop.edu   

Cook Children’s Medical Center Recruiting
Fort Worth, Texas, United States, 76104
Contact: Dianna Grado    682-885-2844    Dianna.grado@cookchildrens.org   

Praxis Precision Medicines

PRAX-562

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 encephaolopathies (DEEs), including SCN8A-DEE.
PRAX-562 demonstrated robust preclinical ant-seizure activity and exhibited an improved preclinical tolerability compared to standard of care.

Longboard Pharmaceuticals

Now Recruiting – 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.