A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
Dravet Syndrome (DS)
About this trial
This is an interventional treatment trial for Dravet Syndrome (DS) focused on measuring Drug Therapy
Eligibility Criteria
Inclusion Criteria:
- Has documented clinical diagnosis of DS.
- Had ≥12 convulsive seizures over 12 weeks before screening based on the historical information and has had ≥4 convulsive seizures per 28 days during the 4- to 6-week prospective baseline period.
- Weighs ≥10 kg at the screening visit (Visit 1).
- Failure to control seizures despite appropriate trials of at least 1 ASM based on historical information and is currently on an antiseizure therapy or other treatment options considered as SOC.
- Artisanal cannabidiols are allowed at a stable dose for at least 4 weeks before the screening visit (Visit 1); the dosing regimen and manufacturer should remain constant throughout the study (Artisanal cannabidiols will not be counted as ASMs.).
- Currently taking 0 to 4 ASMs at stable doses for at least 4 weeks before the screening visit (Visit 1); benzodiazepines used chronically (daily) to treat seizures are considered ASMs. Fenfluramine and cannabidiol (Epidiolex) are allowed where available and should be counted as an ASM. ASM dosing regimen must remain constant throughout the study.
Exclusion Criteria:
1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.
Sites / Locations
- Phoenix Childrens HospitalRecruiting
- David Geffen School of Medicine at UCLARecruiting
- University of California Benioff Children's HospitalRecruiting
- Pediatric Neurology PARecruiting
- Clinical Integrative Research Center of AtlantaRecruiting
- University of Iowa Hospitals & Clinics - (CRS)Recruiting
- NYU Comprehensive Epilepsy CenterRecruiting
- University of ToledoRecruiting
- Oregon Health and Science UniversityRecruiting
- Medical University of South CarolinaRecruiting
- Cook Children's Medical Center
- Seattle Children's HospitalRecruiting
- Multicare Health System - Mary Bridge PediatricsRecruiting
- Sydney Children's HospitalRecruiting
- Queensland Childrens HospitalRecruiting
- Austin HospitalRecruiting
- Alfred HospitalRecruiting
- UZ Antwerpen PINRecruiting
- Hopital Universitaire des Enfants Reine FabiolaRecruiting
- Instituto de Neurologia de Curitiba (INC)
- Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
- Hospital das Clinicas - UNICAMP
- Universidade Federal de Sao Paulo
- Universidade de Sao PauloRecruiting
- Alberta Childrens HospitalRecruiting
- Child and Family Research InstituteRecruiting
- Hospital For Sick ChildrenRecruiting
- Peking University First HospitalRecruiting
- Beijing Children's Hospital,Capital Medical UniversityRecruiting
- Children's Hospital of Chongqing Medical University
- The Second Affiliated Hospital of Guangzhou Medical UniversityRecruiting
- Guangzhou Women And Children's Medical CenterRecruiting
- Shenzhen Children's HospitalRecruiting
- Wuhan Childrens hospitalRecruiting
- Xiangya Hospital Central South UniversityRecruiting
- The First Hospital of Jilin UniversityRecruiting
- Children's Hospital of ShanghaiRecruiting
- Children's Hospital of Fudan UniversityRecruiting
- CHRU Dijon Hopital General
- Hopital Roger SalengroRecruiting
- Hopitaux de La TimoneRecruiting
- Hopital Necker - Enfants MaladesRecruiting
- Hopital Robert DebreRecruiting
- Schon Klinik VogtareuthRecruiting
- Klinikum der Johann-Wolfgang Goethe-UniversitatRecruiting
- Krankenhaus Mara gGmbH - Epilepsiezentrum BethelRecruiting
- Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige GmbhRecruiting
- Childrens' Hospital of Athens 'P. and A. Kyriakou'
- Attikon University General HospitalRecruiting
- University General Hospital of Larissa
- Hippokration Hospital
- Szent Janos Korhaz es Eszak-Budai Egyesitett KorhazakRecruiting
- Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti IntezetRecruiting
- IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PINRecruiting
- Fondazione Policlinico Universitario A GemelliRecruiting
- ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCSRecruiting
- Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PINRecruiting
- Aichi Medical University HospitalRecruiting
- Fukuoka Children's HospitalRecruiting
- Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical CenterRecruiting
- Kumamoto-Ezuko Medical Center for The Severely DisabledRecruiting
- National Hospital Organization Nagasaki Medical CenterRecruiting
- National Hospital Organization Nishi-Niigata Chuo National HospitalRecruiting
- Okayama University HospitalRecruiting
- Yasuhara Childrens ClinicRecruiting
- Osaka City General HospitalRecruiting
- Osaka University HospitalRecruiting
- National Hospital Organization Shizuoka Institute of Epilepsy and Neurological DisordersRecruiting
- Hokkaido University HospitalRecruiting
- National Center of Neurology and PsychiatryRecruiting
- Childrens University HospitalRecruiting
- Kempenhaeghe - PPDSRecruiting
- Stichting Epilepsie Instellingen NederlandRecruiting
- Centrum Medyczne PlejadyRecruiting
- Neurosphera SP. Z O.ORecruiting
- Uniwersyteckie Centrum Kliniczne
- Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w PoznaniuRecruiting
- Russian National Research Medical University n.a. N.I.Pirogov
- UGMK-Zdorojie, LLC
- Krasnoyarsk State Medical University n.a. V.F. Voyno-Ysenetskiy
- Russian National Research Medical University n.a. N.I.Pirogov
- Tyumen State Medical Academy
- Clinic for Neurology and Psychiatry for Children and YouthRecruiting
- Mother and Child Health Care Institute of Serbia Dr Vukan CupicRecruiting
- University Clinical Center NisRecruiting
- Children and Youth Health Care Institute of VojvodinaRecruiting
- Clinica Universidad Navarra
- Hospital Universitario Vall d'Hebron - PPDSRecruiting
- Hospital Regional Universitario de Malaga Hospital GeneralRecruiting
- Centro de Neurologia AvanzadaRecruiting
- Hospital Universitari i Politecnic La Fe de ValenciaRecruiting
- Municipal Institution Dnipropetrovsk Regional Children Clinical Hospital of DRC
- Communal Non-profit Enterprise City Childrens Clinical Hospital #6 of DCC
- Communal Non-commercial Enterprise Iv-Frank Regional Childrens Clinical Hosp of Iv-Frank RC
- CNPE Clinical Hospital Psychiatry of the Executive Body of the Kyiv City Council KCSA
- SI Ukr. Med. Rehabilitation Center For Children With Organic Injury of Nervous System of MoH of Ukr
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Soticlestat
Placebo
Participants weighing <45kg: Soticlestat, mini-tablets, at the dose of 40mg to 200mg, orally or via gastrostomy tube (G-tube) or low-profile gastric tube (MIC-KEY button) or jejunostomy tube (J-tube), twice daily (BID) based on the body weight up to 4 weeks in Titration Period. Participants will continue to receive dose that they are on at the end of Titration Period, for 12 weeks in Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45kg: Soticlestat mini-tablets or tablets with a starting dose of 100mg BID followed by 200 mg BID and, then 300mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment.
Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or MIC-KEY button or J-tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks. Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.