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NCT06872125

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

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-04

Study Completion Date

2027-08-31

Brief Summary

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

Detailed Description

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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 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.

Conditions

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Dravet Syndrome

Keywords

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Pediatric epilepsy Epileptic Encephalopathies Refractory Myoclonic Epilepsy Severe Myoclonic Epilepsy in Infancy STK-001

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Zorevunersen

Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham

Group Type EXPERIMENTAL

zorevunersen

Intervention Type DRUG

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

Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham

Group Type SHAM_COMPARATOR

Sham Comparator

Intervention Type OTHER

Sham group will not have drug administered. Sham group will have a lumbar puncture with CSF withdrawal.

Interventions

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zorevunersen

Intervention Type DRUG

Sham Comparator

Sham group will not have drug administered. Sham group will have a lumbar puncture with CSF withdrawal.

Intervention Type OTHER

Eligibility Criteria

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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 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.

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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Responsible Party

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Responsibility Role SPONSOR

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Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

STK-001-DS-301

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Zorevunersen

zorevunersen

Sham Comparator

Sham Comparator

Interventions

zorevunersen

Sham Comparator

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Stoke Therapeutics, Inc

Responsible Party

Locations

Nationwide Children's Hospital

Oregon Health & Science University (OHSU)

Children's Hospital of Philadelphia

LeBonheur Children's Hospital

Cook Children's Medical Center

Texas Children's Hospital

UVA Health

Seattle Children's Hospital

Fukuoka Children's Hospital

Hokkaido University Hospital

Kyoto University Hospital

Nagoya University Hospital

National Hospital Organization Nishi Niigata Central Hospital

Okayama University Hospital

Jichi Medical University Hospital

National Center of Neurology and Psychiatry

Yokohama City University Medical Center

Royal Hospital for Children

Phoenix Children's Hospital

Arkansas Children's Hospital

Cedars Sinai Medical Center

Children's Hospital of Orange County

USCF Medical Center

Children's Hospital Colorado

Children's National Medical Center

Nemours Children's Health

Nicklaus Children's Hospital

Advent Health Neuroscience Research Institute

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

University of Iowa Hospital and Clinics

Massachusetts General Hospital

Boston Children's Hospital

CS Mott Children's Hospital

Mayo Clinic

Washington University in St. Louis School of Medicine

NYU Langone Health

Weill Cornell Medicine

University of Rochester Medical Center

University of North Carolina at Chapel Hill

Duke University Health System

Cincinnati Children's Hospital Medical Center

Cleveland Clinic

Great Ormond Street Hospital for Children

Sheffield Children's Hospital

Countries

Central Contacts

Emperor Information Center

Facility Contacts

Nigel Negm

Angus Wilfong, MD

Debopam Samanta, MD

Andrea Sellew

Research Assistant

Maija-Riikka Steenari, MD

Kaitlyn Sherer

Antoinette Swanson

Ebonee Hayes