Summary

Eligibility
for people ages 2-18 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion

Description

Summary

Stoke Therapeutics is evaluating the safety and tolerability of single ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency and quality of life will be measured as secondary endpoints in this open-label study.

Official Title

An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents With Dravet Syndrome

Details

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.

Keywords

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

Eligibility

You can join if…

Open to people ages 2-18

  • Must have DS with onset prior to 12 months of age with recurrent focal motor or hemiconvulsive or generalized tonic-clonic seizures, which are often prolonged and triggered by hyperthermia.
  • No history of causal MRI lesion
  • No other known etiology
  • Normal development at seizure onset.
  • Documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene associated with DS.
  • Had at least 2 treatments for epilepsy that either had lack of adequate seizure control (requiring an additional AED) or had to be discontinued due to an AE(s).
  • Currently be taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening.
  • All epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) must have been stable (including product type, dose, and setting) for at least 4 weeks prior to Screening.

You CAN'T join if...

  • Known pathogenic mutation in another gene that causes epilepsy
  • Currently being treated with an antiepileptic drug acting primarily as a sodium channel blocker including: phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
  • Clinically significant unstable medical conditions other than epilepsy.
  • Has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Screening or prior to dosing on Day 1, other than epilepsy.
  • Any other significant disease or disorder which, in the opinion of the Investigator, may either put the patient at risk because of participation in the study, may influence the results of the study, or may affect the patient's ability to participate in the study.

Locations

  • University of California San Francisco Medical Center accepting new patients
    San Francisco California 94158 United States
  • Oregon Health & Science University accepting new patients
    Portland Oregon 97239 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Stoke Therapeutics, Inc
ID
NCT04442295
Phase
Phase 1/2
Study Type
Interventional
Last Updated