Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
Tuberous Sclerosis Complex
About this trial
This is an interventional treatment trial for Tuberous Sclerosis Complex focused on measuring Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive
Eligibility Criteria
Inclusion Criteria: Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001. Participant/parent(s)/LAR(s) willing and able to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures. If the participant is not qualified or able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide assent for study participation, if appropriate. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study. Willing and able to take Investigational product (IP) (suspension) as directed with food TID. Women of childbearing potential (WOCBP) must be using a medically acceptable method of birth control and have a negative quantitative serum beta-human chorionic growth hormone (β-HCG) test collected at the initial visit. Childbearing potential is defined as a female who is biologically capable of becoming pregnant. Medically acceptable methods of birth control include intrauterine devices (that have been in place for at least 1 month prior to the screening visit), hormonal contraceptives (eg, combined oral contraceptives, patch, vaginal ring, injectables, and implants), and surgical sterilization (such as oophorectomy or tubal ligation). When used consistently and correctly, "double-barrier" methods of contraception can be used as an effective alternative to highly effective contraception methods. Contraceptive measures such as Plan B™, sold for emergency use after unprotected sex, are not acceptable methods for routine use Male participants must agree to use highly effective contraceptive methods during the study and for 30 days after the last dose of IP. Highly effective methods of contraception include surgical sterilization (such as a vasectomy) and adequate "double-barrier" methods. Exclusion Criteria: Pregnant or breastfeeding. An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease. History of psychogenic nonepileptic seizures. Any disease or condition (other than TSC) at the initial visit that could compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy. This may include any illness in the past 4 weeks which in the opinion of the investigator may affect seizure frequency. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study. Have active suicidal plan/intent or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 6 months. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
Sites / Locations
- Barrow Neurological Institute at Phoenix Children's Hospital
- Arkansas Children's Research Institute
- UCLA Mattel Children's Hospital, TSC Center
- Children's Hospital of Orange County
- Children's Hospital Colorado
- Nemours Children's Hospital - Delaware Valley
- University of Florida Gainsville
- NW FL Clinical Research Group, LLC
- Nicklaus Children's Hospital
- Comprehensive Neurology Clinic
- Children's Healthcare of Atlanta
- Ann & Robert H. Lurie Children's Hospital of Chicago
- Mid-Atlantic Epilepsy & Sleep Center
- Boston Children's Hospital, Harvard Medical School
- Children's Hospital of Michigan Central Michigan University
- Mayo Clinic - Rochester
- Children's Mercy Hospital
- TSC Clinic at Northeast Regional Epilepsy Group
- Institute of Neurology and Neurosurgery at Saint Barnabas
- University of Rochester Medical Center
- University of North Carolina at Chapel Hill
- Atrium Health/Levine Children's Hospital
- Duke University Medical Center
- Cincinnati Children's Hospital Medical Center
- Penn State Children's Hospital
- Children's Hospital of Philadelphia
- Medical University of South Carolina
- Le Bonheur Children's Hospital
- Child Neurology Consultants of Austin (CNCA)
- University of Texas Southwestern Medical Center
- McGovern Medical School at the University of Texas Health Science Center
- University of Utah Health Care-Pediatric Neurology
- Seattle Children's Hospital
- Queensland Health
- Austin Health
- Royal Brisbane and Women's Hospital
- Alfred Health
- Western Sydney Local health District
- Royal Melbourne Hospital
- The Royal Children's Hospital Melbourne
- Prince of Wales Hospital
- Antwerp University Hospital (UZA)
- UZ Brussel
- Hôtel Dieu de Montréal - CHUM
- CHU Sainte-Justine
- The Hospital for Sick Children
- Toronto Western Hospital
- BC Children's Hospital
- Peking University First Hospital
- The Affiliated Hospital of Guizhou Medical University
- University Hospital of Lyon
- University Hospital of Lille
- Robert-Debré Hospital
- Hôpital de la Pitié-Salpêtrière
- University Hospital of Rennes
- University of Strasbourg
- Epilepsie-Zentrum Bethel - Krankenhaus Mara
- University Hospital Bonn
- ZNN - Epilepsiezentrum Frankfurt am Main
- Universitäts Krankenhaus Freiburg
- Gemeinschaftskrankenhaus Herdecke
- Epilepsiezentrum Kleinwachau gGmbH
- Soroka University Medical Center
- Hadassah Medical Center
- Schneider Children´s Medical Center
- Tel-Aviv Sourasky Medical Center
- Sheba Medical Center
- Department of Neurology and Sense Organs, AOU Policlinico di Bari
- Azienda Ospedaliero-Universitaria Meyer
- Pediatric Neurology and Muscular Diseases Unit - University of Genoa
- Children's Hospital Bambino Gesù
- Policlinico Umberto I
- Hospital de la Santa Creu i Sant Pau
- Hospital Universitari Vall d'Hebron
- Hospital Sant Joan de Déu
- Hospital Infantil Universitario Niño Jesús
- Hospital Ruber International
- Hospital Regional Universitario de Málaga
- Hospital Universitario y Politécnico La Fe
- Royal Aberdeen Children's Hospital, NHS Grampian
- Bristol Royal Hospital for Children
- Leeds General Infirmary
- NHS acute tertiary referral centre, John Radcliffe Hospital
- Salford Royal Hospital
- Sheffield Children's Hospital
Arms of the Study
Arm 1
Experimental
Ganaxolone (GNX) oral suspension, 3 times a day (TID)