Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
NCT ID: NCT05604170
Last Updated: 2025-10-01
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
117 participants
INTERVENTIONAL
2022-05-16
2025-04-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Ganaxolone (GNX) oral suspension, 3 times a day (TID)
Ganaxolone
GNX will be administered.
Interventions
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Ganaxolone
GNX will be administered.
Eligibility Criteria
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Inclusion Criteria
2. 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.
3. Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study.
4. Willing and able to take Investigational product (IP) (suspension) as directed with food TID.
5. 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
6. 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
2. An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease.
3. History of psychogenic nonepileptic seizures.
4. 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.
5. Unwillingness to avoid excessive alcohol use or cannabis use throughout the study.
6. 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.
7. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
8. Exposed to any other investigational drug (except for GNX in Study 1042-TSC-2001 or Study 1042-TSC-3001) or investigational device within 30 days or fewer than 5 half-lives prior to Visit 1 (first visit of the OLE). For therapies in which half-life cannot be readily established, the Sponsor's medical monitor should be consulted.
1 Year
65 Years
ALL
No
Sponsors
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Marinus Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Arkansas Children's Research Institute
Little Rock, Arkansas, United States
UCLA Mattel Children's Hospital, TSC Center
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Hospital - Delaware Valley
Wilmington, Delaware, United States
Boston Children's Hospital, Harvard Medical School
Boston, Massachusetts, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Children's Mercy Hospital
Kansas City, Missouri, 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
Atrium Health/Levine Children's Hospital
Charlotte, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Child Neurology Consultants of Austin (CNCA)
Austin, Texas, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
McGovern Medical School at the University of Texas Health Science Center
Houston, Texas, United States
University of Utah Health Care-Pediatric Neurology
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Austin Health
Heidelberg, , Australia
Royal Brisbane and Women's Hospital
Herston, , Australia
Alfred Health
Melbourne, , Australia
Royal Melbourne Hospital
Parkville, , Australia
Hôtel Dieu de Montréal - CHUM
Montreal, , Canada
CHU Sainte-Justine
Montreal, , Canada
The Hospital for Sick Children
Toronto, , Canada
Toronto Western Hospital
Toronto, , Canada
BC Children's Hospital
Vancouver, , Canada
Peking University First Hospital
Beijing, , China
Beijing Children Hospital, Capital Medical University
Beijing, , China
Chinese PLA General Hospital
Beijing, , China
The Affiliated Hospital of Guizhou Medical University
Beijing, , China
First Hospital of Jilin University
Jilin, , China
University Hospital of Lyon
Bron, , France
Hôpital Sud
Rennes, , France
University of Strasbourg
Strasbourg, , France
Epilepsie-Zentrum Bethel - Krankenhaus Mara
Bielefeld, , Germany
University Hospital Bonn
Bonn, , Germany
ZNN - Epilepsiezentrum Frankfurt am Main
Frankfurt, , Germany
Universitäts Krankenhaus Freiburg
Freiburg im Breisgau, , Germany
Gemeinschaftskrankenhaus Herdecke
Herdecke, , Germany
Epilepsiezentrum Kleinwachau gGmbH
Radeberg, , Germany
Schneider Children´s Medical Center
Petah Tikva, , Israel
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
Genova, , Italy
Policlinico Umberto I
Rome, , Italy
Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Sant Joan de Déu
Barcelona, , Spain
Hospital Infantil Universitario Niño Jesús
Madrid, , Spain
Hospital Ruber International
Madrid, , Spain
Hospital Regional Universitario de Málaga
Málaga, , Spain
Hospital Universitario y Politécnico La Fe
Valencia, , Spain
Bristol Royal Hospital for Children
Bristol, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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1042-TSC-3002
Identifier Type: -
Identifier Source: org_study_id
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