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New hope for kids with severe epilepsy: drug trial targets seizures

NCT ID NCT06872125

Disease control Sponsor: Stoke Therapeutics, Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Aug 25, 2026 · Updated 7 times

Summary

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living skills.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
zorevunersen (STK-001)
What this could lead to
If successful, this could provide a new treatment option to reduce seizures and improve quality of life for children with Dravet syndrome.
What could go wrong
This is an early-stage Phase 3 trial with 170 participants, so results may not apply to all patients. The drug is given via spinal injection, which carries risks like headache or infection.
Why investors are watching

Stoke Therapeutics is running a phase 3 trial of zorevunersen, a drug for Dravet syndrome, a severe form of epilepsy. For a small company, this readout is the main event: a positive result could support approval, while a failure would leave the company without a clear path forward.

If it works: If the trial shows the drug reduces seizures safely, Stoke could move closer to bringing the first treatment for Dravet syndrome to market. That outcome would validate the company's approach and likely attract partners or funding.

If it fails: Phase 3 trials fail often, and a negative or unclear result could delay or end the program. For a small company with few other products, that could sharply reduce its value and make it harder to raise money.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 170 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dravet syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    3 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Advent Health Neuroscience Research Institute

    ACTIVE_NOT_RECRUITING

    Orlando, Florida, 32804, United States

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    ACTIVE_NOT_RECRUITING

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    ACTIVE_NOT_RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    ACTIVE_NOT_RECRUITING

    Florence, Italy

  • Boston Children's Hospital

    ACTIVE_NOT_RECRUITING

    Boston, Massachusetts, 02115, United States

  • CS Mott Children's Hospital

    ACTIVE_NOT_RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Cedars Sinai Medical Center

    ACTIVE_NOT_RECRUITING

    Los Angeles, California, 90048, United States

  • Charité - Campus Virchow-Klinikum

    ACTIVE_NOT_RECRUITING

    Berlin, Germany

  • Children's Hospital Colorado

    ACTIVE_NOT_RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    ACTIVE_NOT_RECRUITING

    Orange, California, 92868, United States

  • Children's National Medical Center

    ACTIVE_NOT_RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Cleveland Clinic

    ACTIVE_NOT_RECRUITING

    Cleveland, Ohio, 44195, United States

  • Clinica Universidad de Navarra

    ACTIVE_NOT_RECRUITING

    Pamplona, Spain

  • Cook Children's Medical Center

    ACTIVE_NOT_RECRUITING

    Fort Worth, Texas, 76104, United States

  • Duke University Health System

    ACTIVE_NOT_RECRUITING

    Durham, North Carolina, 27705, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    ACTIVE_NOT_RECRUITING

    Roma, Italy

  • Fukuoka Children's Hospital

    ACTIVE_NOT_RECRUITING

    Fukuoka, Japan

  • Great Ormond Street Hospital for Children

    ACTIVE_NOT_RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hokkaido University Hospital

    ACTIVE_NOT_RECRUITING

    Hokkaido, Japan

  • Hospital Blua Sanitas Valdebebas

    ACTIVE_NOT_RECRUITING

    Madrid, Spain

  • Hospital Ruber Internacional

    ACTIVE_NOT_RECRUITING

    Madrid, Spain

  • Hôpital Necker - Enfants Malades

    ACTIVE_NOT_RECRUITING

    Paris, France

  • Hôpital Robert Debré - Paris

    ACTIVE_NOT_RECRUITING

    Paris, France

  • Hôpital de la Timone

    ACTIVE_NOT_RECRUITING

    Marseille, France

  • Integriertes Sozialpaediatrisches Zentrum

    ACTIVE_NOT_RECRUITING

    München, Germany

  • Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS

    ACTIVE_NOT_RECRUITING

    Genova, Italy

  • Jichi Medical University Hospital

    ACTIVE_NOT_RECRUITING

    Shimotsuke, Japan

  • Kyoto University Hospital

    ACTIVE_NOT_RECRUITING

    Kyoto, Japan

  • LeBonheur Children's Hospital

    ACTIVE_NOT_RECRUITING

    Memphis, Tennessee, 38103, United States

  • Massachusetts General Hospital

    ACTIVE_NOT_RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    ACTIVE_NOT_RECRUITING

    Rochester, Minnesota, 55905, United States

  • NHO Shizuoka

    ACTIVE_NOT_RECRUITING

    Shizuoka, Japan

  • NYU Langone Health

    ACTIVE_NOT_RECRUITING

    New York, New York, 10016, United States

  • Nagoya University Hospital

    ACTIVE_NOT_RECRUITING

    Nagoya, Japan

  • National Center of Neurology and Psychiatry

    ACTIVE_NOT_RECRUITING

    Tokyo, Japan

  • National Hospital Organization Nishi Niigata Central Hospital

    ACTIVE_NOT_RECRUITING

    Niigata, Japan

  • Nationwide Children's Hospital

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43205, United States

  • Nemours Children's Health

    ACTIVE_NOT_RECRUITING

    Jacksonville, Florida, 32207, United States

  • Nicklaus Children's Hospital

    ACTIVE_NOT_RECRUITING

    Miami, Florida, 33155, United States

  • Okayama University Hospital

    ACTIVE_NOT_RECRUITING

    Okayama, Japan

  • Oregon Health & Science University (OHSU)

    ACTIVE_NOT_RECRUITING

    Portland, Oregon, 97239, United States

  • Osaka City General Hospital

    ACTIVE_NOT_RECRUITING

    Osaka, Japan

  • Ospedale Pediatrico Bambino Gesù

    ACTIVE_NOT_RECRUITING

    Roma, Italy

  • Peking University First Hospital

    RECRUITING

    Beijing, China

  • Phoenix Children's Hospital

    ACTIVE_NOT_RECRUITING

    Phoenix, Arizona, 85016, United States

  • Royal Hospital for Children

    ACTIVE_NOT_RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • Sheffield Children's Hospital

    ACTIVE_NOT_RECRUITING

    Sheffield, S10 2TH, United Kingdom

  • Texas Children's Hospital

    ACTIVE_NOT_RECRUITING

    Houston, Texas, 77030, United States

  • The Second Affiliated Hospital of Guangzhou Medical university

    RECRUITING

    Guangzhou, China

  • USCF Medical Center

    ACTIVE_NOT_RECRUITING

    San Francisco, California, 94158, United States

  • UVA Health

    ACTIVE_NOT_RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Universitaetsklinikum Bonn AoeR

    ACTIVE_NOT_RECRUITING

    Bonn, Germany

  • Universitaetsklinikum Frankfurt Goethe-Universitaet

    ACTIVE_NOT_RECRUITING

    Frankfurt, Germany

  • Universitaetsklinikum Freiburg

    ACTIVE_NOT_RECRUITING

    Friedberg, Germany

  • Universitaetsklinikum Heidelberg

    ACTIVE_NOT_RECRUITING

    Heidelberg, Germany

  • University of North Carolina at Chapel Hill

    ACTIVE_NOT_RECRUITING

    Chapel Hill, North Carolina, 27514, United States

  • University of Rochester Medical Center

    ACTIVE_NOT_RECRUITING

    Rochester, New York, 14642, United States

  • University of Utah Primary Children's Hospital

    ACTIVE_NOT_RECRUITING

    Salt Lake City, Utah, 84113, United States

  • Weill Cornell Medicine

    ACTIVE_NOT_RECRUITING

    New York, New York, 10021, United States

  • Wuhan Children's Hospital

    RECRUITING

    Wuhan, China

  • Yokohama City University Medical Center

    ACTIVE_NOT_RECRUITING

    Yokohama, Japan

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