The Cute Syndrome Foundation: SCN8A Epilepsy Support, Awareness, and Research
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SCN8A Clinical Trials

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.

SEE IF YOU OR YOUR CHILD MAY QUALIFY

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! 

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.
KAYAK STUDY FAQS
SENTINEL COHORT FAQS
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
VIEW THE STUDY ON CLINICALTRIALS.GOV

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.

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.
PRE-SCREENING INFORMATION

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.
VIEW THE PRESS RELEASE

Educational Webinars

Kayak Study Update: September 2022
Embold Study Update: September 2022
View more from our Clinical Trial Readiness virtual series!
Past Annual Reports

Past PCDH19 Efforts

​Privacy Policy

The Cute Syndrome Foundation

PO Box 842 Ozark, MO 65721​
The Cute Syndrome is registered as a tax-exempt organization under IRS section 501(c)(3).
​
Our tax identification number is: 46-2699066.

© The Cute Syndrome Foundation, All rights reserved
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  • Home
  • About
    • About SCN8A
    • About Us
    • Partner Organizations
    • Volunteers
    • Sponsors
    • 2021 Annual Report
  • Families
    • Caregiver Support
    • CUTE Connections Grant Application
    • Join our SCN8A Community
    • SCN8A Warriors
    • Educational Videos
    • Reference Guide
    • Resources
    • Shareable Graphics
  • Events
    • Annual SCN8A Clinician, Researcher, and Family Gathering
    • Researcher Roundtable
    • Awareness Day
    • Virtual 5k
  • Research
    • Clinical Trials
    • Research Grants
    • SCN8A Registry
  • Donate
  • Contact
  • Blog
  • Shop