RECRUITING

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

Conditions

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

EMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients With Dravet Syndrome

Quick Facts

Study Start:2025-06

Study Completion:2027-08

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06872125

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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: 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.	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.

Inclusion Criteria

Exclusion 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:
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.

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.

Contacts and Locations

Study Contact

Emperor Information Center

CONTACT

1-781-430-8200

info@emperorstudy.com

Study Locations (Sites)

Phoenix Children's Hospital

Phoenix, Arizona, 85016

United States

Arkansas Children's Hospital

Little Rock, Arkansas, 72202

United States

Cedars Sinai Medical Center

Los Angeles, California, 90048

United States

Children's Hospital of Orange County

Orange, California, 92868

United States

USCF Medical Center

San Francisco, California, 94158

United States

Children's Hospital Colorado

Aurora, Colorado, 80045

United States

Children's National Medical Center

Washington D.C., District of Columbia, 20010

United States

Nemours Children's Health

Jacksonville, Florida, 32207

United States

Nicklaus Children's Hospital

Miami, Florida, 33155

United States

Advent Health Neuroscience Research Institute

Orlando, Florida, 32804

United States

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

Chicago, Illinois, 60611

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Boston Children's Hospital

Boston, Massachusetts, 02115

United States

CS Mott Children's Hospital

Ann Arbor, Michigan, 48109

United States

Mayo Clinic

Rochester, Minnesota, 55905

United States

Washington University in St. Louis School of Medicine

St. Louis, Missouri, 63110

United States

NYU Langone Comprehensive Epilepsy Center

New York, New York, 10016

United States

Weill Cornell Medicine

New York, New York, 10021

United States

University of Rochester Medical Center

Rochester, New York, 14642

United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514

United States

Duke University Health System

Durham, North Carolina, 27705

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

United States

Cleveland Clinic

Cleveland, Ohio, 44195

United States

Nationwide Children's Hospital

Columbus, Ohio, 43205

United States

Oregon Health & Science University (OHSU)

Portland, Oregon, 97239

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

LeBonheur Children's Hospital

Memphis, Tennessee, 38103

United States

Cook Children's Medical Center

Fort Worth, Texas, 76104

United States

Texas Children's Hospital

Houston, Texas, 77030

United States

UVA Health

Charlottesville, Virginia, 22903

United States

Seattle Children's Hospital

Seattle, Washington, 98105

United States

Collaborators and Investigators

Sponsor: Stoke Therapeutics, Inc

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06

Study Completion Date2027-08

Study Record Updates

Study Start Date2025-06

Study Completion Date2027-08

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

Dravet Syndrome