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Looking for participantsPhase3

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

Sponsor: Stoke Therapeutics, Inc

NCT ID: NCT06872125

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
zorevunersen (drug), Sham Comparator (other)
How long the study runs
Study runs about 40 months (dates as stated)
About the drug or intervention
zorevunersen — drug: Treatment Period 1: Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281. · Sham Comparator — other: Treatment Period 1: Sham group will not have drug administered.
Patient visit burden
Not specified by the sponsor

In plain English

This study is testing a medicine called zorevunersen in children and teenagers aged 2 to under 18 with Dravet syndrome, a severe form of epilepsy. Researchers want to find out how well it works, and how safe and tolerable it is. The study is run by Stoke Therapeutics, Inc.

Who can take part

  • Aged 2 to under 18 years
  • Has a clinical diagnosis of Dravet syndrome confirmed by the Epilepsy Study Consortium, Inc. (ESCI), with seizures starting before 13 months of age and no other known cause
  • Has a change (variant) in the SCN1A gene that is known or thought to be harmful, or is of uncertain significance — a negative SCN1A test means the person cannot join
  • Has a set number of major motor seizures (such as tonic-clonic, hemiclonic, or drop attacks) during a 6-week observation period
  • Has tried at least 2 previous treatments for seizures, such as anti-seizure medicines (ASMs), a ketogenic diet, or vagus nerve stimulation (VNS)
  • Is taking at least one anti-seizure medicine (ASMs) regularly, including regular benzodiazepines
  • All current seizure medicines and other treatments (including cannabis- or cannabinoid-based products) must have been kept steady during the baseline period, unless the dose changed with weight

Who may not be able to

  • Has an SCN1A gene change known as 'gain-of-function'
  • Is taking a regular anti-seizure medicine that mainly works as a sodium channel blocker (for example phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate)
  • Uses certain brain stimulation treatments (responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation) — vagus nerve stimulation (VNS) is allowed
  • Developed a new seizure type, or an old seizure type came back, during the baseline period, or had more than 1 hospital stay for seizures during that period

What taking part involves

  • • Taking the study medicine zorevunersen or a dummy medicine (placebo), as this is a double-blind study — meaning neither you nor the study team knows which one you are given
  • • Not stated — ask the trial team about how the medicine is given, the dose, and how long treatment lasts

Time commitment: Taking part involves a 6-week observation period and a baseline period before treatment, plus study visits — full details of the number of visits and total study length are not stated — ask the trial team.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
2 Years to 17 Years
Who
All
Number of participants
170
Started
2025-06-04
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for dravet syndrome
  • • Phase3 - 170 participants
  • • The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome

Who can take part?

  • • Ages 2 Years to 17 Years
  • • Diagnosed with dravet syndrome

Where?

  • • Glasgow - Royal Hospital for Children
  • • London - Great Ormond Street Hospital for Children
  • • Sheffield - Sheffield Children's Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.

More detail

Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome. It 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. This is a global, multicenter, randomized, double-blind, sham-controlled, parallel group Phase 3 study to assess the efficacy, safety, and tolerability of zorevunersen in patients with Dravet syndrome. The study duration and endpoints are designed to evaluate the potential of zorevunersen for disease modification. The study consists of two parts, Treatment Period 1 and Treatment Period 2. The primary and secondary endpoints will be assessed at the conclusion of Treatment Period 1. These endpoints will be assessed again at the end of Treatment Period 2. The primary endpoint is the change from baseline in major motor seizure frequency. Secondary endpoints include the change in behavior and cognition, clinical status, and health-related quality of life in patients with Dravet syndrome. Patients will have the opportunity to enroll in an open label extension study and receive zorevunersen if they meet eligibility criteria at the end of the study.

Dravet Syndrome

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 2 Years - 17 Years
  • Who can join: All genders

Biomarkers mentioned

results of Negative

Treatment history

Treatments you must have had:

  • ✓ a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc
  • ✓ number of major motor seizures during the 6-week Observation Period
  • ✓ used at least 2 prior interventions for seizures
  • ✓ been stable (unless adjusted for weight) during the Baseline Period

What the study is looking for

  • ✓Patients must be ≥2 and \<18 years of age.
  • ✓Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by:
  • ✓Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis (i.e., not as needed...

Who cannot take part

  • ✗Patient has documented variant in the SCN1A gene associated with gain-of-function
  • ✗Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain...
See the full criteria
Key Inclusion Criteria: 1. Patients must be ≥2 and \<18 years of age. 2. Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months (inclusive, \<13 months), of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures. No other known etiology causing clinical DS manifestations.. 3. Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the sodium voltage-gated channel type 1 alpha subunit (SCN1A) gene. Patients who have SCN1A testing results of Negative (no variants identified) cannot be randomized. 4. Patient must experience the required number of major motor seizures during the 6-week Observation Period. Major motor seizure types included are Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic. 5. Patient must have used at least 2 prior interventions for seizures. These can include anti-seizure medications (ASMs), ketogenic diet and/or vagus nerve stimulation (VNS) with either lack of adequate seizure control or discontinued due to an AE(s). These interventions can be ongoing therapies. 6. Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis (i.e., not as needed \[PRN\]) for any indication will be considered an ASM. 7. Patients' maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS), as well as any marijuana- or cannabinoid-based products, must have been stable (unless adjusted for weight) during the Baseline Period. Key Exclusion Criteria: 1. Patient has documented variant in the SCN1A gene associated with gain-of-function 2. Patient is currently treated with a maintenance ASM acting primarily as a sodium channel blocker, including but not limited to phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate, given the mechanism of action of zorevunersen. 3. Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS. 4. Patient has emergence of a new seizure type or reemergence of a past seizure type (seizure types that last occurred more than 12 months before Screening Visit A) during the Baseline Period, or has more than 1 hospitalization for seizures during the Baseline Period.

Where Is This Study? (3 UK sites)

Royal Hospital for Children

Glasgow G51 4TF, United Kingdom

ACTIVE_NOT_RECRUITING

Great Ormond Street Hospital for Children

London WC1N 3JH, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Sheffield Children's Hospital

Sheffield S10 2TH, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Alessia Dunn

STH.ResearchAdministration@nhs.net0114 2712550

How to Get in Touch

Emperor Information Center

Sponsor contact

CONTACT

1-781-430-8200 info@emperorstudy.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-08