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 age 2-21. This is the first SCN8A specific clinical trial and is a huge step forward for our community!
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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 medinfo@neurocrine.com 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
Cook Children's Hospital—Fort Worth, TX Cook Children's Medical Center Fort Worth, Texas, United States, 76104 Contact: Dianna Grado, 682-885-2844 Dianna.grado@cookchildrens.org University of California San Francisco—San Francisco, CA UCSF Medical Center San Francisco, California, United States, 94158 Contact: Lucy Liu, 415-353-2437 Lucy.liu3@ucsf.edu Children's National—Washington, DC Children's National Hospital Washington, District of Columbia, United States, 20010 Contact: Zachary Kramer zkramer@childrensnational.org Children's Hospital of Philadelphia—Philadelphia, PA Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States, 19104 Contact: Jennie Minnick ENGIN@chop.edu |
University of Rochester—Rochester, NY
University of Rochester Rochester, New York, United States, 14642 Contact: Noreen Connolly Noreen_Connolly@urmc.rochester.edu Ann & Robert H. Lurie Children's Hospital of Chicago—Chicago, IL Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States, 60611 Contact: Enrique Rojas erojas@luriechildrens.org Not Yet Recruiting Wake Forest Baptist Health—Winston-Salem, NC Wake Forest Baptist Health Winston-Salem, North Carolina, United States, 27157 Contact: Carolyn Hedrick cwhedric@wakehealth.edu University of Utah—Salt Lake City, UT University of Utah Salt Lake City, Utah, United States, 84132 Contact: Carly Straley clinicaltrialsoffice@hsc.utah.edu |
Praxis Precision Medicines: PRAX-562
Embold Study
Praxis Precision Medicines' SCN8A study is now pre-screening.
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.
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 participants 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.
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.
Educational Webinars
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Kayak Study Update: September 2022
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Embold Study Update: September 2022
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