A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
NCT ID: NCT06872125
Last Updated: 2026-02-09
Study Results
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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RECRUITING
PHASE3
170 participants
INTERVENTIONAL
2025-06-04
2028-10-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Zorevunersen
Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham in Treatment Period 1 (approximately 52 weeks). Upon the completion of Treatment Period 1 all eligible patients, will enter Treatment Period 2 and receive zorevunersen, regardless of initial treatment assignment.
zorevunersen
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.
Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84).
Sham Comparator
Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham
Sham Comparator
Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration.
Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84).
Interventions
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zorevunersen
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.
Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84).
Sham Comparator
Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration.
Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84).
Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
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.
2 Years
17 Years
ALL
No
Sponsors
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Stoke Therapeutics, Inc
INDUSTRY
Responsible Party
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Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Cedars Sinai Medical Center
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
USCF Medical Center
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nemours Children's Health
Jacksonville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Advent Health Neuroscience Research Institute
Orlando, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
CS Mott Children's Hospital
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University in St. Louis School of Medicine
St Louis, Missouri, United States
NYU Langone Health
New York, New York, United States
Weill Cornell Medicine
New York, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Duke University Health System
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health & Science University (OHSU)
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
LeBonheur Children's Hospital
Memphis, Tennessee, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Utah Primary Children's Hospital
Salt Lake City, Utah, United States
UVA Health
Charlottesville, Virginia, United States
Fukuoka Children's Hospital
Fukuoka, , Japan
Hokkaido University Hospital
Hokkaido, , Japan
Kyoto University Hospital
Kyoto, , Japan
Nagoya University Hospital
Nagoya, , Japan
National Hospital Organization Nishi Niigata Central Hospital
Niigata, , Japan
Okayama University Hospital
Okayama, , Japan
Osaka City General Hospital
Osaka, , Japan
Jichi Medical University Hospital
Shimotsuke, , Japan
NHO Shizuoka
Shizuoka, , Japan
National Center of Neurology and Psychiatry
Tokyo, , Japan
Yokohama City University Medical Center
Yokohama, , Japan
Royal Hospital for Children
Glasgow, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Angus Wilfong, MD
Role: backup
Andrea Sellew
Role: backup
Research Assistant
Role: primary
Ryan Kennington
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Research Assistant
Role: primary
Other Identifiers
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2024-519555-28
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
STK-001-DS-301
Identifier Type: -
Identifier Source: org_study_id
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