An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Dravet Syndrome Clinical Trial

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

Status Enrolling by invitation

Clinical Trial Summary

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.

Clinical Trial Description

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. ;

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Syndrome

• NCT number: NCT04740476
• Study type: Interventional
• Source: Stoke Therapeutics, Inc
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: January 21, 2021
• Completion date: March 3, 2027