A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Start Date
6/4/2025
Completion Date
10/1/2028
Summary
The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
Detailed Description
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 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.
Eligibility Criteria
Age Range: 2 years to 17 years
Interventions
zorevunersen
Sham Comparator
Conditions
Locations
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
NYU Langone Health
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
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
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
University of Utah Primary Children's Hospital
Salt Lake City, Utah 84113
United States
UVA Health
Charlottesville, Virginia 22903
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, G51 4TF
United Kingdom
Great Ormond Street Hospital for Children
London, WC1N 3JH
United Kingdom
Sheffield Children's Hospital
Sheffield, S10 2TH
United Kingdom