A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Recruiting · NCT06872125 · Interventional (participants receive a specific treatment) · Lead sponsor: Stoke Therapeutics, Inc
View the official record on ClinicalTrials.gov →Interventions studied
Sham Comparatorzorevunersen
What this trial is about
The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
Who can take part
Age range
2 Years to 17 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
Inclusion criteria
- Patients must be ≥2 and \<18 years of age.
- Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by:
- nset, 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..
- 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.
- 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.
- 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.
- 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.
- 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.
- Key
Exclusion criteria
- Patient has documented variant in the SCN1A gene associated with gain-of-function
- 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.
- Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS.
- 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.
Where it is running
60 locations listed across 18 US states.
- 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
- Peking University First Hospital - Beijing, China
- Charité - Campus Virchow-Klinikum - Berlin, Germany
- Universitaetsklinikum Bonn AoeR - Bonn, Germany
- Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer - Florence, Italy
- Universitaetsklinikum Frankfurt Goethe-Universitaet - Frankfurt, Germany
- Universitaetsklinikum Freiburg - Friedberg, Germany
- Fukuoka Children's Hospital - Fukuoka, Japan
- Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS - Genova, Italy
- Royal Hospital for Children - Glasgow, United Kingdom
- The Second Affiliated Hospital of Guangzhou Medical university - Guangzhou, China
- Universitaetsklinikum Heidelberg - Heidelberg, Germany
- Hokkaido University Hospital - Hokkaido, Japan
- Kyoto University Hospital - Kyoto, Japan
- Great Ormond Street Hospital for Children - London, United Kingdom
- Hospital Blua Sanitas Valdebebas - Madrid, Spain
- Hospital Ruber Internacional - Madrid, Spain
- Hôpital de la Timone - Marseille, France
- Integriertes Sozialpaediatrisches Zentrum - München, Germany
- 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
- Clinica Universidad de Navarra - Pamplona, Spain
- Hôpital Necker - Enfants Malades - Paris, France
- Hôpital Robert Debré - Paris - Paris, France
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Roma, Italy
- Ospedale Pediatrico Bambino Gesù - Roma, Italy
- Sheffield Children's Hospital - Sheffield, United Kingdom
- Jichi Medical University Hospital - Shimotsuke, Japan
- NHO Shizuoka - Shizuoka, Japan
- National Center of Neurology and Psychiatry - Tokyo, Japan
- Wuhan Children's Hospital - Wuhan, China
Showing 60 of 61 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →