Participating in clinical trials is an important way to help accelerate therapeutic development. There are currently two SCN8A studies in various phases. Find more information about each study below.
KAYAK STUDY (Neurocrine) | EMBOLD STUDY (Praxis)
Neurocrine Biosciences: NBI-921352
Kayak Study
Neurocrine Biosciences' SCN8A study is now enrolling.
This is a study to evaluate NBI-921352 as adjunctive therapy in subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) in patients aged 2-21. This is the first SCN8A-specific clinical trial and is a huge step forward for our community! Estimated completion: December 2025
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SENTINEL COHORT:
The DEE2012 “KAYAK” study will enroll in two parts: a Sentinel Cohort of 8 participants and a Main Cohort of up to 52 participants. There will be a pause in participants screening and enrollment for data analysis by an independent data monitoring committee (DMC) and any needed protocol amendments following the full enrollment of the Sentinel Cohort. |
To learn more about this study, you or your doctor may contact the study research staff:
Neurocrine Medical Information Call Center 877-641-3461 [email protected] ClinicalTrials.gov identifier (NCT number): NCT04873869 |
Neurocrine Trial Sites
(Currently USA only, international enrollment anticipated in 2023)
Travel Support: If an individual qualifies to participate in this clinical trial, study-related medical exams and study-related laboratory tests will be performed at no cost to them. Compensation for travel or lodging support may also be available. For sites that have agreed to use it, a travel service is being provided to assist with booking flights, rail, car rentals, taxis, wheelchair vans, lodging, etc.
Travel Support: If an individual qualifies to participate in this clinical trial, study-related medical exams and study-related laboratory tests will be performed at no cost to them. Compensation for travel or lodging support may also be available. For sites that have agreed to use it, a travel service is being provided to assist with booking flights, rail, car rentals, taxis, wheelchair vans, lodging, etc.
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Recruiting
San Francisco, CA
University of California San Francisco UCSF Medical Center San Francisco, California, United States, 94158 Contact: Lucy Liu, 415-353-2437 [email protected] Washington, DC Children's National Hospital Washington, District of Columbia, United States, 20010 Contact: Zachary Kramer [email protected] Chicago, IL Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States, 60611 Contact: Enrique Rojas [email protected] Rochester, MN Mayo Clinic Rochester, Minnesota, United States, 55905 Contact: Bridget Neja, 507-266-9150 [email protected] Rochester, NY University of Rochester Rochester, New York, United States, 14642 Contact: Noreen Connolly [email protected] |
Philadelphia, PA Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Contact: Jennie Minnick [email protected] Fort Worth, TX Cook Children's Medical Center Fort Worth, Texas, United States, 76104 Contact: Dianna Grado, 682-885-2844 [email protected] Not Yet Recruiting
Winston-Salem, NC
Wake Forest Baptist Health Winston-Salem, North Carolina, United States, 27157 Contact: Carolyn Hedrick [email protected] Salt Lake City, UT University of Utah Salt Lake City, Utah, United States, 84132 Contact: Carly Straley [email protected] |
Praxis Precision Medicines: PRAX-562
Embold Study
Sept. 2024: Praxis Precision Medicines has announced positive topline results from the Embold study
Emboldstudy.com is live, allowing the opportunity for caregivers of those diagnosed with SCN2A and SCN8A DEE to see the inclusion and exclusion criteria and schedule a pre-screening call with a nurse navigator. This provides the caregiver with access to a live person to assist them and answer questions. The information of those who qualify will be shared with the study site. Estimated completion: June 2025
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The EMBOLD Study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety, tolerability, efficacy, and pharmacokinetics of PRAX-562 in pediatric patients age 2-18 with DEEs, followed by an open-label extension.
PRAX-562 is a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in early onset SCN2A-DEE and SCN8A-DEE. |
To learn more about this study, you or your doctor may contact the study research staff:
Head of Pharmacovigilance 617-300-8460 [email protected] ClinicalTrials.gov identifier (NCT number): NCT05818553 |
Praxis Trial Sites
(USA and International)
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Recruiting
Atlanta, GA Praxis Research Site Atlanta, Georgia, United States, 30329 Chicago, IL Praxis Research Site Chicago, Illinois, United States, 60611 Boston, MA Praxis Research Site Boston, Massachusetts, United States, 02115 |
Tacoma, WA Praxis Research Site Tacoma, Washington, United States, 98405 Madrid, Spain Praxis Research Site Madrid, Spain, 28034 Fully remote from your home [email protected] |
Educational Webinars
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Kayak Study Update: September 2022
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Embold Study Update: September 2022
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Kayak Study Update: April 2023 (update at 5:30 mark)
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Embold Study Update: April 2023
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