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NCT Number: NCT04740476

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

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Dravet Syndrome Brain Diseases Central Nervous System Diseases Epilepsies, Myoclonic Epilepsy Epilepsy, Generalized Epileptic Syndromes Nervous System Diseases

Age range

30 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of California San Francisco Medical Center, San Francisco, California, United States

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About this study

This study is a multi-center, open-label, multiple-dose, safety extension study for patients who have completed another study of zorevunersen and meet study eligibility criteria. zorevunersen is an investigational new medicine for the treatment of Dravet syndrome. Zorevunersen 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).

Zorevunersen 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 zorevunersen.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Completed dosing with zorevunersen 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.

Exclusion criteria

  • 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 or rufinamide (with the exception of cenobamate, which is permitted).
  • 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 zorevunersen) 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.

Treatment and study plan

zorevunersen (STK-001)

Drug

zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.

Primary outcomes

  1. Safety of multiple doses of zorevunersen

    Time frame: Screening (Day -1) until 6 months after multiple drug dosing

    Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and outcomes on the cerebellar function clinical screening battery.

Secondary outcomes

  1. Pharmacokinetic (PK) Parameters

    Time frame: Dosing (Day 1) until 6 months after multiple drug dosing

    Analysis of plasma concentrations of zorevunersen

  2. Exposure of zorevunersen in Cerebrospinal Fluid (CSF)

    Time frame: Dosing (Day 1) and every 4 months until last study drug dosing day

    Measurement of zorevunersen concentrations

  3. Measurement of Seizure Frequency

    Time frame: Screening (Day -1) until 6 months after multiple drug dosing

    Measurement of Seizure Frequency (by paper diary)

  4. Change in overall clinical status

    Time frame: Screening (Day -1) until 6 months after multiple drug dosing

    Change in overall clinical status as measured by the Clinical Global Impression of Change (CGIC) and the Caregiver Global Impression of Change (CaGIC)

  5. Change in Quality of Life

    Time frame: Screening (Day -1) until 6 months after multiple drug dosing

    Change in quality of life as measured by the EuroQoL-five dimensions, youth version (EQ-5D-Y) instrument

Sponsors and collaborators

Lead sponsor

Stoke Therapeutics, Inc

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2029
Study completion
2029
First posted
Feb 5, 2021
Registry last updated
Feb 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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