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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
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
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
Where Is This Study? (3 UK sites)
Royal Hospital for Children
Glasgow G51 4TF, United Kingdom
Great Ormond Street Hospital for Children
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Sheffield Children's Hospital
Sheffield S10 2TH, United Kingdom
How to Get in Touch
Emperor Information Center
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