for people ages 30 months and up (full criteria)
at San Francisco, California and other locations
study started
completion around



Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

Official Title

An Open-Label Extension Study for Patients With Dravet Syndrome Who Previously Participated in Studies of STK-001


This study is a multi-center, open-label, multiple-dose, safety extension study for patients who have completed another study of STK-001 and meet study eligibility criteria. STK-001 is an investigational new medicine for the treatment of Dravet syndrome. STK-001 is an antisense oligonucleotide (ASO) that is intended to increase the level of productive SCN1A messenger RNA (mRNA) and consequently increase the expression of the sodium channel Nav1.1 protein. This RNA-based approach is not gene therapy, but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA).

STK-001 is designed to upregulate Nav1.1 protein expression from the nonmutant (wild-type) copy of the SCN1A gene to restore physiological Nav1.1 levels. Nav1.1 levels are reduced in people with Dravet syndrome. Stoke has generated preclinical data demonstrating proof-of-mechanism for STK-001.


Dravet Syndrome, Pediatric epilepsy, Epileptic Encephalopathies, Refractory Myoclonic Epilepsy, Severe Myoclonic Epilepsy in Infancy, Myoclonic Epilepsies, Syndrome, STK-001


You can join if…

Open to people ages 30 months and up

  • Completed dosing with STK-001 and the End of Study Visit in Study STK-001-DS-101 or Study STK-001-DS-102, with an acceptable safety profile per Investigator judgment.
  • Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 or Study STK-001-DS-102 per Investigator and Sponsor judgment.
  • Completed Study STK-001-DS-101 or STK-001-DS-102 within 4 weeks of the start of their participation in Study STK-001-DS-501 unless approved by sponsor.

You CAN'T join if...

  • Met any withdrawal criteria from Study STK-001-DS-101 or STK-001-DS-102.
  • Currently treated with an antiepileptic drug (AED) acting primarily as a sodium channel blocker, as maintenance therapy, including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide or cenobamate.
  • Clinically significant unstable medical conditions other than epilepsy.
  • Clinically relevant symptoms or a clinically significant illness (in the judgment of the Investigator) at Screening or prior to dosing on Day 1, other than epilepsy.
  • Spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt.
  • Treated (or is being treated) with an investigational product (other than STK-001) since participating in Study STK-001-DS-101 or STK-001-DS-102.
  • Participating in an observational study, they are excluded unless approved by the Sponsor.


  • UCSF Medical Center
    San Francisco California 94158 United States
  • Oregon Health & Science University
    Portland Oregon 97239 United States


accepting new patients by invitation only
Start Date
Completion Date
Stoke Therapeutics, Inc
Phase 2 research study
Study Type
Expecting 60 study participants
Last Updated