RecruitingPhase 3NCT06872125

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

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


Sponsor

Stoke Therapeutics, Inc

Enrollment

170 participants

Start Date

Jun 4, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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


Eligibility

Min Age: 2 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Sham Comparator and a drug called zorevunersen for people with dravet syndrome. The study is currently recruiting participants at 61 locations. People eligible for this study include aged 2 Years to 17 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGzorevunersen

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).

OTHERSham 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).


Locations(58)

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

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

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

Cleveland Clinic

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Oregon Health & Science University (OHSU)

Portland, Oregon, 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

Hôpital de la Timone

Marseille, France

Hôpital Necker - Enfants Malades

Paris, France

Hôpital Robert Debré - Paris

Paris, France

Charité - Campus Virchow-Klinikum

Berlin, Germany

Universitaetsklinikum Bonn AoeR

Bonn, Germany

Universitaetsklinikum Frankfurt Goethe-Universitaet

Frankfurt, Germany

Universitaetsklinikum Freiburg

Friedberg, Germany

Universitaetsklinikum Heidelberg

Heidelberg, Germany

Integriertes Sozialpaediatrisches Zentrum

München, Germany

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, Italy

Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS

Genova, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

Ospedale Pediatrico Bambino Gesù

Roma, Italy

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

Hospital Blua Sanitas Valdebebas

Madrid, Spain

Hospital Ruber Internacional

Madrid, Spain

Clinica Universidad de Navarra

Pamplona, Spain

Royal Hospital for Children

Glasgow, United Kingdom

Great Ormond Street Hospital for Children

London, United Kingdom

Sheffield Children's Hospital

Sheffield, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06872125


Related Trials