Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
NCT ID: NCT05323734
Last Updated: 2025-07-11
Study Results
Results available
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
Recruitment Status
COMPLETED
Clinical Phase
PHASE3
Total Enrollment
129 participants
Study Classification
INTERVENTIONAL
Study Start Date
2022-04-01
Study Completion Date
2024-10-14
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective Baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a 4-week titration period (Day 1 to Day 28) and a 12-week maintenance period (Day 29 to Week 16).
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
NCT05604170
Tuberous Sclerosis Complex
TERMINATED
PHASE3
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
NCT04285346
Tuberous Sclerosis
COMPLETED
PHASE2
Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old
NCT05249556
CDKL5 Deficiency Disorder
NOT_YET_RECRUITING
PHASE3
Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
NCT03350035
Status Epilepticus
Convulsive Status EPILEPTICUS
Non Convulsive Status Epilepticus
+1 more
COMPLETED
PHASE2
Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension
NCT01963208
Drug Resistant Partial Onset Seizure
COMPLETED
PHASE3
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Tuberous Sclerosis Complex
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
Allocation Method
RANDOMIZED
Intervention Model
PARALLEL
Primary Study Purpose
TREATMENT
Blinding Strategy
TRIPLE
Participants
Caregivers
Investigators
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Ganaxolone (GNX)
oral suspension, 3 times a day (TID)
Group Type
EXPERIMENTAL
Ganaxolone
Intervention Type
DRUG
GNX will be administered
Placebo matching GNX
oral suspension, TID
Group Type
PLACEBO_COMPARATOR
Placebo
Intervention Type
DRUG
Placebo will be administered
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Ganaxolone
GNX will be administered
Intervention Type
DRUG
Placebo
Placebo will be administered
Intervention Type
DRUG
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
1. Clinical or mutational diagnosis of TSC consistent with:
1. Molecular confirmation of a pathogenic mutation in TSC1 or TSC2. A pathogenic mutation is defined as a mutation that clearly prevents protein synthesis and/or inactivates the function of the TSC1 or TSC2 proteins (eg, nonsense mutation or frameshift mutations, large genomic deletions) or is a missense mutation whose effect on protein function has been established by functional assessment. The Principal investigator (PI) or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR
2. Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features.
2. Male or female participants aged 1 through 65 years, inclusive. For Europe (EU), Middle East and North Africa (MENA), and Oceania (OC) Male or Female participants aged 2 through 65 years, inclusive.
3. Participant/parent(s) or LAR(s) willing 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 nor able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide consent for study participation, if appropriate.
4. Failure to control seizures despite appropriate trial of 2 or more Anti-seizure medication (ASMs) at therapeutic doses and for adequate duration of treatment per PI judgment.
5. Participants should be on a stable regimen of ASMs (including moderate or strong inducer or inhibitor ASM eg, carbamazepine, phenytoin, etc.) at therapeutic doses for ≥ 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis and it should be discussed with the study medical monitor.)
6. A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month. This includes seizures of any kind.
7. Have at least 8 primary endpoint seizures in the first 28 days following the screening visit.
The primary endpoint seizure types are defined as the following:
1. focal motor seizures without impairment of consciousness or awareness
2. focal seizures with impairment of consciousness or awareness with motor features
3. focal seizures evolving to bilateral, tonic-clonic seizures
4. generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures.
Seizures that do not count towards the primary endpoint include:
1. Focal or generalized nonmotor seizures (eg, absence seizures or focal nonmotor seizures with or without impairment of awareness)
2. Infantile or epileptic spasms
3. Myoclonic seizures.
8. Participants with surgically implanted vagal nerve stimulator (VNS) will be allowed to enter the study provided that all of the following conditions are met:
1. The VNS has been in place for ≥ 6 months prior to the screening visit.
2. The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study.
3. The battery is expected to last for the duration of the study.
9. Parent(s)/caregiver(s)/LAR(s) or the participant, as appropriate, is (are) willing and able to maintain an accurate and complete daily seizure eDiary for the duration of the study.
10. Willing and able to take IP (suspension) as directed with food (TID).
11. 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 screening and Baseline visits.Childbearing potential is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes 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.
12. 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.
1. Molecular confirmation of a pathogenic mutation in TSC1 or TSC2. A pathogenic mutation is defined as a mutation that clearly prevents protein synthesis and/or inactivates the function of the TSC1 or TSC2 proteins (eg, nonsense mutation or frameshift mutations, large genomic deletions) or is a missense mutation whose effect on protein function has been established by functional assessment. The Principal investigator (PI) or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR
2. Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features.
2. Male or female participants aged 1 through 65 years, inclusive. For Europe (EU), Middle East and North Africa (MENA), and Oceania (OC) Male or Female participants aged 2 through 65 years, inclusive.
3. Participant/parent(s) or LAR(s) willing 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 nor able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide consent for study participation, if appropriate.
4. Failure to control seizures despite appropriate trial of 2 or more Anti-seizure medication (ASMs) at therapeutic doses and for adequate duration of treatment per PI judgment.
5. Participants should be on a stable regimen of ASMs (including moderate or strong inducer or inhibitor ASM eg, carbamazepine, phenytoin, etc.) at therapeutic doses for ≥ 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis and it should be discussed with the study medical monitor.)
6. A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month. This includes seizures of any kind.
7. Have at least 8 primary endpoint seizures in the first 28 days following the screening visit.
The primary endpoint seizure types are defined as the following:
1. focal motor seizures without impairment of consciousness or awareness
2. focal seizures with impairment of consciousness or awareness with motor features
3. focal seizures evolving to bilateral, tonic-clonic seizures
4. generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures.
Seizures that do not count towards the primary endpoint include:
1. Focal or generalized nonmotor seizures (eg, absence seizures or focal nonmotor seizures with or without impairment of awareness)
2. Infantile or epileptic spasms
3. Myoclonic seizures.
8. Participants with surgically implanted vagal nerve stimulator (VNS) will be allowed to enter the study provided that all of the following conditions are met:
1. The VNS has been in place for ≥ 6 months prior to the screening visit.
2. The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study.
3. The battery is expected to last for the duration of the study.
9. Parent(s)/caregiver(s)/LAR(s) or the participant, as appropriate, is (are) willing and able to maintain an accurate and complete daily seizure eDiary for the duration of the study.
10. Willing and able to take IP (suspension) as directed with food (TID).
11. 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 screening and Baseline visits.Childbearing potential is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes 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.
12. 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
1. Previous exposure to GNX.
2. Pregnant or breastfeeding.
3. Participants who have been taking felbamate for less than 1 year prior to screening.
4. Participants taking cannabidiol (CBD) preparations other than Epidiolex.
5. A positive result on plasma drug screen for CBD or tetrahydrocannabinol (THC) at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which can be adjusted by the investigator in the event of any Adverse events (AEs).
6. Concurrent use of adrenocorticotropic hormone (ACTH), prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of cytochrome P450 3A4 (CYP3A4), rifampin and St John's Wort. Participants on ACTH, prednisone, or other systemically (non-inhaled or topical) administered steroids should be off the product \> 28 days prior to screening. Rifampin and St John's Wort must be discontinued at least 28 days before Visit 2, study drug initiation.
Note:
1. Use of concomitant intranasal or pro re nata (PRN) topical steroids for dermatologic reactions and allergic rhinitis are allowed during the study.
2. This exclusion criterion does not prohibit the use of approved ASMs.
7. Changes in any chronic medications within the 4 weeks prior to the screening visit. All chronic concomitant medications must be relatively stable in dose for at least 4 weeks prior to the screening visit unless otherwise noted. Small dose adjustment to manage tolerability and safety events is permitted and should be discussed with the study medical monitor.
8. Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening.
9. An active central nervous system (CNS) infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain magnetic resonance imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control.
10. Any disease or condition (medical or surgical; other than TSC) at the screening visit that might 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.
11. Hepatic impairment sufficient to affect participant safety, or an aspartate aminotransferase (AST)/ serum glutamic oxaloacetic transaminase (SGOT) or alanine aminotransferase (ALT)/ serum glutamic pyruvic transaminase (SGPT) \> 3 × the upper limit of normal (ULN) at screening or Baseline visits and confirmed by a repeat test.
12. Biliary impairment sufficient to affect participant safety, or total bilirubin levels \> 1.5 × ULN at screening or Baseline visit and confirmed by a repeat test. In cases of Gilbert's Syndrome, resulting in stable levels of total bilirubin greater than ULN, the medical monitor can determine if a protocol exception can be made
13. Renal impairment sufficient to affect participant safety, or estimated glomerular filtration rate (eGFR) \< 30 milliliter per minute (mL/min) (calculated using the Cockcroft-Gault formula or Pediatric GFR calculator or Bedside Schwartz), will be excluded from study entry or will be discontinued if the criterion is met post Baseline. Cases of temporary renal insufficiency should be discussed with the medical monitor to determine the participant's study continuation.
14. Exposed to any other investigational drug or investigational device within 30 days or fewer than 5 half-lives prior to the screening visit. For therapies in which half-life cannot be readily established, the Sponsor's Medical Monitor should be consulted.
15. Unwillingness to avoid excessive alcohol use throughout the study.
16. Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months.
17. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
18. Participants deprived of their liberty by a judicial or administrative decision, or for psychiatric treatment, or participants admitted to a health or social services facility for purposes other than research.
19. Participants receiving traditional Chinese medicine therapies within the prior 28 days of the screening.
2. Pregnant or breastfeeding.
3. Participants who have been taking felbamate for less than 1 year prior to screening.
4. Participants taking cannabidiol (CBD) preparations other than Epidiolex.
5. A positive result on plasma drug screen for CBD or tetrahydrocannabinol (THC) at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which can be adjusted by the investigator in the event of any Adverse events (AEs).
6. Concurrent use of adrenocorticotropic hormone (ACTH), prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of cytochrome P450 3A4 (CYP3A4), rifampin and St John's Wort. Participants on ACTH, prednisone, or other systemically (non-inhaled or topical) administered steroids should be off the product \> 28 days prior to screening. Rifampin and St John's Wort must be discontinued at least 28 days before Visit 2, study drug initiation.
Note:
1. Use of concomitant intranasal or pro re nata (PRN) topical steroids for dermatologic reactions and allergic rhinitis are allowed during the study.
2. This exclusion criterion does not prohibit the use of approved ASMs.
7. Changes in any chronic medications within the 4 weeks prior to the screening visit. All chronic concomitant medications must be relatively stable in dose for at least 4 weeks prior to the screening visit unless otherwise noted. Small dose adjustment to manage tolerability and safety events is permitted and should be discussed with the study medical monitor.
8. Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening.
9. An active central nervous system (CNS) infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain magnetic resonance imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control.
10. Any disease or condition (medical or surgical; other than TSC) at the screening visit that might 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.
11. Hepatic impairment sufficient to affect participant safety, or an aspartate aminotransferase (AST)/ serum glutamic oxaloacetic transaminase (SGOT) or alanine aminotransferase (ALT)/ serum glutamic pyruvic transaminase (SGPT) \> 3 × the upper limit of normal (ULN) at screening or Baseline visits and confirmed by a repeat test.
12. Biliary impairment sufficient to affect participant safety, or total bilirubin levels \> 1.5 × ULN at screening or Baseline visit and confirmed by a repeat test. In cases of Gilbert's Syndrome, resulting in stable levels of total bilirubin greater than ULN, the medical monitor can determine if a protocol exception can be made
13. Renal impairment sufficient to affect participant safety, or estimated glomerular filtration rate (eGFR) \< 30 milliliter per minute (mL/min) (calculated using the Cockcroft-Gault formula or Pediatric GFR calculator or Bedside Schwartz), will be excluded from study entry or will be discontinued if the criterion is met post Baseline. Cases of temporary renal insufficiency should be discussed with the medical monitor to determine the participant's study continuation.
14. Exposed to any other investigational drug or investigational device within 30 days or fewer than 5 half-lives prior to the screening visit. For therapies in which half-life cannot be readily established, the Sponsor's Medical Monitor should be consulted.
15. Unwillingness to avoid excessive alcohol use throughout the study.
16. Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months.
17. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
18. Participants deprived of their liberty by a judicial or administrative decision, or for psychiatric treatment, or participants admitted to a health or social services facility for purposes other than research.
19. Participants receiving traditional Chinese medicine therapies within the prior 28 days of the screening.
Minimum Eligible Age
1 Year
Maximum Eligible Age
65 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Marinus Pharmaceuticals
INDUSTRY
Sponsor Role
lead
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Responsibility Role
SPONSOR
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Arkansas Children's Research Institute
Little Rock, Arkansas, United States
Site Status
UCLA Mattel Children's Hospital, TSC Center
Los Angeles, California, United States
Site Status
Children's Hospital of Orange County
Orange, California, United States
Site Status
University of California, San Diego
San Diego, California, United States
Site Status
Childrens Hospital Colorado
Aurora, Colorado, United States
Site Status
Nemours Children's Hospital - Delaware Valley
Wilmington, Delaware, United States
Site Status
University of Florida Gainesville
Gainesville, Florida, United States
Site Status
Nemours Children's Health
Jacksonville, Florida, United States
Site Status
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, United States
Site Status
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Site Status
Children's Mercy Hosptial
Kansas City, Missouri, United States
Site Status
University of Rochester Medical Center
Rochester, New York, United States
Site Status
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Site Status
Atrium Health/Levine Children's Hospital
Charlotte, North Carolina, United States
Site Status
Duke University Medical Center
Durham, North Carolina, United States
Site Status
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Site Status
Penn State Children's Hospital
Hershey, Pennsylvania, United States
Site Status
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Site Status
Child Neurology Consultants of Austin (CNCA)
Austin, Texas, United States
Site Status
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Site Status
McGovern Medical School at the University of Texas Health Science Center
Houston, Texas, United States
Site Status
University of Utah Health Care-Pediatric Neurology
Salt Lake City, Utah, United States
Site Status
Seattle Children's Hospital
Seattle, Washington, United States
Site Status
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia
Site Status
Austin Health
Heidelberg, , Australia
Site Status
Alfred Health
Melbourne, , Australia
Site Status
Royal Melbourne Hospital
Parkville, , Australia
Site Status
Hôtel Dieu de Montréal - CHUM
Montreal, , Canada
Site Status
CHU Sainte-Justine
Montreal, , Canada
Site Status
The Hospital for Sick Children
Toronto, , Canada
Site Status
Toronto Western Hospital
Toronto, , Canada
Site Status
BC Children's Hospital
Vancouver, , Canada
Site Status
First Hospital of Jilin University
Changchun, Jilin, China
Site Status
Jiangxi Provincial Children's Hospital
Jiangxi, Nanchang City, China
Site Status
Beijing Children Hospital, Capital Medical University
Beijing, Xicheng District, China
Site Status
The Affiliated Hospital of Guizhou Medical University
Guiyang, Yunyan District, China
Site Status
Peking University First Hospital
Beijing, , China
Site Status
Chinese PLA General Hospital
Beijing, , China
Site Status
Chengdu's Women and Children's Central Hospital
Chengdu, , China
Site Status
University Hospital of Lyon
Bron, , France
Site Status
University Hospital of Rennes
Rennes, , France
Site Status
University of Strasbourg
Strasbourg, , France
Site Status
Epilepsie-Zentrum Bethel - Krankenhaus Mara
Bielefeld, , Germany
Site Status
University Hospital Bonn
Bonn, , Germany
Site Status
ZNN - Epilepsiezentrum Frankfurt am Main
Frankfurt, , Germany
Site Status
Universitäts Krankenhaus Freiburg
Freiburg im Breisgau, , Germany
Site Status
Gemeinschaftskrankenhaus Herdecke
Herdecke, , Germany
Site Status
Epilepsiezentrum Kleinwachau gGmbH
Radeberg, , Germany
Site Status
Schneider Children´s Medical Center
Petah Tikva, , Israel
Site Status
Sheba Medical Center
Tel Litwinsky, , Israel
Site Status
Department of Neurology and Sense Organs, AOU Policlinico di Bari
Bari, , Italy
Site Status
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
Genova, , Italy
Site Status
Policlinico Umberto I
Rome, , Italy
Site Status
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
Site Status
Hospital Sant Joan de Déu
Barcelona, , Spain
Site Status
Hospital Infantil Universitario Niño Jesús
Madrid, , Spain
Site Status
Hospital Ruber International
Madrid, , Spain
Site Status
Hospital Regional Universitario de Málaga
Málaga, , Spain
Site Status
Hospital Universitario y Politécnico La Fe
Valencia, , Spain
Site Status
Bristol Royal Hospital for Children
Bristol, , United Kingdom
Site Status
NHS acute tertiary referral centre, John Radcliffe Hospital
Oxford, , United Kingdom
Site Status
Salford Royal Hospital
Salford, , United Kingdom
Site Status
Sheffield Children's Hospital
Sheffield, , United Kingdom
Site Status
Countries
Review the countries where the study has at least one active or historical site.
United States
Australia
Canada
China
France
Germany
Israel
Italy
Spain
United Kingdom
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
1042-TSC-3001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.
Randomized Therapy In Status Epilepticus
NCT04391569
COMPLETED
PHASE3
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
NCT03572933
COMPLETED
PHASE3
Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
NCT02358538
COMPLETED
PHASE2
A Randomized, Controlled Trial of Ganaxolone in Adult Uncontrolled Partial-Onset Seizures
NCT00465517
COMPLETED
PHASE2
To Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus (RSE)
NCT05814523
WITHDRAWN
PHASE3
Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE)
NCT05757544
WITHDRAWN
PHASE2
A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures
NCT02519439
TERMINATED
PHASE3
Open-label Extension to Protocol 1042-0600
NCT00512317
COMPLETED
PHASE2
A Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601
NCT01002820
COMPLETED
PHASE2
An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures
NCT06908356
RECRUITING
PHASE2
A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
NCT04938427
COMPLETED
PHASE3
Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Cenobamate Adjunctive Therapy in PGTC Seizures
NCT03678753
COMPLETED
PHASE3
Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex
NCT02098759
UNKNOWN
NA
A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures
NCT01713946
COMPLETED
PHASE3
A Efficacy and Safety Study of Adjunctive Perampanel in Primary Generalized Tonic Clonic Seizures
NCT01393743
COMPLETED
PHASE3
A Study of the Efficacy and Safety of Topiramate as an add-on Therapy in the Treatment of Epilepsy Patients With Lennox-Gastaut Syndrome
NCT00236756
COMPLETED
PHASE3
Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Lennox-Gastaut Syndrome
NCT02318537
WITHDRAWN
PHASE3
Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome
NCT02834793
TERMINATED
PHASE3
GW273225 Therapy Added To Current Seizure Treatments In Patients With Partial Seizures
NCT00567424
WITHDRAWN
PHASE2
Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures
NCT05288283
TERMINATED
PHASE3
Ganaxolone Expanded Access Program Compassionate Use
NCT04678479
NO_LONGER_AVAILABLE
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
NCT03650452
COMPLETED
PHASE2
A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome
NCT03355209
COMPLETED
PHASE3
A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy
NCT01501383
TERMINATED
PHASE2