A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
Lennox Gastaut Syndrome (LGS)
About this trial
This is an interventional treatment trial for Lennox Gastaut Syndrome (LGS) focused on measuring Drug therapy
Eligibility Criteria
Inclusion Criteria:
- Has documented clinical diagnosis of LGS.
- Has had ≥8 MMD seizures each month in the 3 months prior to Screening based on the historical information and has had ≥8 MMD 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 standard of care (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 3 ASMs at stable doses for at least 4 weeks before the Screening Visit (Visit 1); Fenfluramine and cannabidiol (Epidiolex) are allowed where available and counted as an ASM. ASM dosing regimen must remain constant throughout the study.
Exclusion Criteria:
- Admitted to a medical facility and intubated for treatment of status epilepticus 2 or more times in the 3 months immediately before Screening (Visit 1). For the purpose of this exclusion criterion, status is defined as continuous seizure activity lasting longer than 5 minutes or repeated seizures without return to Baseline in between seizures.
- 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.
- Considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or the participant has attempted suicide within 12 months before the Screening Visit (Visit 1). Participants who have positive answers on item numbers 4 or 5 on the Columbia suicide severity rating scale (C-SSRS) before dosing (Visit 2) are excluded. This scale will only be administered to participants aged ≥6 years.
Sites / Locations
- Phoenix Childrens Hospital
- Center For Neurosciences
- David Geffen School of Medicine at UCLA
- University of California Benioff Children's Hospital
- Children's Hospital Colorado.
- Pediatric Neurology PA
- Clinical Integrative Research Center of Atlanta
- University of Iowa Hospitals & Clinics - (CRS)
- Midatlantic Epilepsy and Sleep Center
- Minnesota Epilepsy Group PA
- Institute of Neurology and Neurosurgery at Saint Barnabas, LLC
- Premier Healthcare Inc.
- Oregon Health and Science University
- Children's Hospital of Philadelphia
- Thomas Jefferson University
- St. Christopher's Hospital for Children
- WellSpan Oncology Research
- Medical University of South Carolina Children Hospital - PIN
- Cook Children's Medical Center - Jane and John Justin Neurosciences Center
- University of Utah - Primary Children's Hospital - PPDS
- MultiCare Institute for Research & Innovation (Tacoma)
- Sydney Children's Hospital
- Queensland Childrens Hospital
- Austin Hospital
- Alfred Hospital
- UZ Antwerpen PIN
- Centre Neurologique William Lennox
- Hopital Universitaire des Enfants Reine Fabiola
- Alberta Childrens HospitalRecruiting
- Child and Family Research Institute
- Hospital For Sick Children
- Peking University First Hospital
- Beijing Children's Hospital,Capital Medical University
- Children's Hospital of Chongqing Medical University
- The Second Affiliated Hospital of Guangzhou Medical University
- Guangzhou Women And Children's Medical Center
- Shenzhen Children's Hospital
- Wuhan Childrens hospital
- Xiangya Hospital Central South University
- Jiangxi Provincial Children's Hospital
- Children's Hospital of Shanghai
- Hopital Roger Salengro
- Hopitaux de La Timone
- Hopital Necker - Enfants Malades
- Hopital Robert Debre
- Schon Klinik VogtareuthRecruiting
- Klinikum der Johann-Wolfgang Goethe-UniversitatRecruiting
- Krankenhaus Mara gGmbH - Epilepsiezentrum BethelRecruiting
- Kleinwachau Sachsisches Epilepsiezentrum Radeberg Gemeinnutzige GmbhRecruiting
- Attikon University General Hospital
- University General Hospital of Larissa
- Hippokration Hospital
- Pecsi Tudomanyegyetem
- Szent Janos Korhaz es Eszak-Budai Egyesitett Korhazak
- Bethesda Gyermekkorhaz
- Orszagos Mentalis, Ideggyogyaszati es Idegsebeszeti Intezet
- Ospedale Bellaria
- Fondazione Policlinico Universitario A Gemelli
- ASST di Pavia - Fondazione Istituto Neurologico Mondino IRCCS
- Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
- Aichi Medical University Hospital
- Fukuoka Children's Hospital
- National Hospital Organization Nagasaki Medical Center
- National Hospital Organization Nishi-Niigata Chuo National Hospital
- Okayama University Hospital
- Yasuhara Childrens Clinic
- Osaka City General Hospital
- Osaka University Hospital
- National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
- National Center of Neurology and Psychiatry
- Childrens University Hospital
- Kempenhaeghe - PPDS
- Stichting Epilepsie Instellingen Nederland
- Centrum Medyczne Plejady
- Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
- UGMK-Zdorojie, LLC
- Clinic for Neurology and Psychiatry for Children and Youth
- Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
- University Clinical Center Nis
- Children and Youth Health Care Institute of Vojvodina
- Clinica Universidad Navarra
- Hospital Universitario Vall d'Hebron - PPDS
- Hospital Regional Universitario de Malaga Hospital General
- Centro de Neurologia Avanzada
- Hospital Universitari i Politecnic La Fe de Valencia
- 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 <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, 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 body weight up to 4 weeks in Titration Period. Participants will continue to receive the dose that they are on at the end of the titration period, for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). Dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45 kg: Soticlestat mini-tablets or tablets with a starting dose of 100 mg BID followed by 200 mg BID and, then 300 mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300 mg BID for 12 weeks in the Maintenance Period. Total duration of the treatment will be up to 16 weeks (Treatment Period). 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 (Treatment Period). Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.