A Phase 3 interventional study of Ganaxolone in Tuberous Sclerosis Complex, sponsored by Marinus Pharmaceuticals. Terminated at 54 sites in 10 countries. Open to participants aged 1 Year to 65 Years. Per ClinicalTrials.gov, last updated 2025-10-01.
Sponsored by Marinus Pharmaceuticals (part of Immedica Pharma) · Phase 3, Interventional, and Treatment
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001
115 studies on the registry are indexed under Tuberous Sclerosis; 27 are open to participants now.
This study's enrollment of 117 is above the median of 52 across 73 interventional studies indexed under Tuberous Sclerosis.
Browse Tuberous Sclerosis studies →Marinus Pharmaceuticals is the lead sponsor of 22 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 9 (100%) have results posted.
First submitted before Marinus Pharmaceuticals became part of Immedica Pharma.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Ganaxolone
GNX will be administered.
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Withdrawals and Dose-Reductions Due to AEs
An adverse event (AE) was any untoward medical occurrence in a clinical study patient, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event was any untoward medical occurrence that, at any dose, results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or any other event that requires scientific judgment.
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Vital Parameters
Vital parameters including heart rate, respiration rate, blood pressure, and body temperature were measured in seated position for at least 5 minutes.
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Physical Examinations
Physical examinations included a full physical evaluation of body systems including: General appearance, head (eyes, ears, nose, and throat), cardiovascular, respiratory, gastrointestinal, genitourinary, musculoskeletal, endocrine/metabolic, hematologic/lymphatic, skin, and other systems as appropriate.
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Neurological Examinations
Neurological examinations included an evaluation of: Cranial nerves, motor exam, sensory exam, reflexes, coordination/cerebellar
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Developmental Examinations
Developmental examinations (applicable only to pediatric participants 1 to 17 years of age, inclusive) included an evaluation of speech/language, motor skills, and social skills
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant 12-lead Electrocardiogram (ECG) Findings
Twelve-lead ECGs were performed by the physician using an ECG machine that automatically calculates the heart rate and measure PR, QRS, QT and QTc intervals.
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Hematology Parameters
Blood samples were collected for the analysis of hematology parameters: hemoglobin, hematocrit, erythrocytes, thrombocytes (platelet count). Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Time frame: Week 1 through Week 150
Number of Participants With Clinically Significant Changes in Chemistry Parameters
Blood samples were collected for the analysis of chemistry parameters: blood urea nitrogen (BUN), potassium, alanine aminotransferase (ALT), alkaline phosphatase (ALP)/serum glutamic pyruvic transaminase (SGPT), total bilirubin, creatinine, sodium, aspartate aminotransferase (AST)/serum glutamic oxaloacetic (SGOT), total protein, fasting blood glucose, calcium, carbon dioxide, estimated glomerular filtration rate (eGFR), and chloride
Time frame: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Urinalysis
Urine samples were collected for the analysis of urinalysis parameters: Specific gravity, color, clarity, pH, glucose, protein, blood, nitrite, protein, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick, and microscopic examination (if blood or protein was abnormal)
Time frame: Up to 150 Weeks
Number of Participants (Age Greater Than 17 Years) With Abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. The score ranges from 1 to 5 and higher scores indicate worse symptoms.
Time frame: Up to Week 52
Percent Change From Baseline in 28-day Seizure Frequency During Open Label Extension
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28. Percent change from baseline in 28-day frequency was calculated for each participant by subtracting Baseline 28-day seizure frequency from post-Baseline 28-day seizure frequency, whole divided by Baseline 28-day seizure frequency and multiplied by 100.
Time frame: Baseline (Day 1), Week 52
A total 117 participants entered the OLE phase and had received at least 1 dose of Ganaxolone (GNX). Of these 8 were previously in Study 1042-TCS-2001 and 109 were previously in Study 1042-TCS-3001 and met eligibility criteria for continued treatment.
| Milestone | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Started | 117 |
| Completed | 0 |
| Not completed | 117 |
| Withdrew: Lack of efficacy | 5 |
| Withdrew: Adverse event | 6 |
| Withdrew: Physician decision | 6 |
| Withdrew: Lost to follow-up | 6 |
| Withdrew: Withdrawal by subject | 11 |
| Withdrew: Early study termination by sponsor | 77 |
| Withdrew: Other | 6 |
An adverse event (AE) was any untoward medical occurrence in a clinical study patient, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event was any untoward medical occurrence that, at any dose, results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or any other event that requires scientific judgment.
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Any serious TEAE | 21 |
| Any TEAE | 79 |
| Withdrawals Due to AEs | 6 |
| Dose-Reduction Due to AEs | 16 |
Vital parameters including heart rate, respiration rate, blood pressure, and body temperature were measured in seated position for at least 5 minutes.
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Vital Parameters | 0 |
Physical examinations included a full physical evaluation of body systems including: General appearance, head (eyes, ears, nose, and throat), cardiovascular, respiratory, gastrointestinal, genitourinary, musculoskeletal, endocrine/metabolic, hematologic/lymphatic, skin, and other systems as appropriate.
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Physical Examinations | 0 |
Neurological examinations included an evaluation of: Cranial nerves, motor exam, sensory exam, reflexes, coordination/cerebellar
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Neurological Examinations | 0 |
Developmental examinations (applicable only to pediatric participants 1 to 17 years of age, inclusive) included an evaluation of speech/language, motor skills, and social skills
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Developmental Examinations | 0 |
Twelve-lead ECGs were performed by the physician using an ECG machine that automatically calculates the heart rate and measure PR, QRS, QT and QTc intervals.
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant 12-lead Electrocardiogram (ECG) Findings | 0 |
Blood samples were collected for the analysis of hematology parameters: hemoglobin, hematocrit, erythrocytes, thrombocytes (platelet count). Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Hematology Parameters | 0 |
Blood samples were collected for the analysis of chemistry parameters: blood urea nitrogen (BUN), potassium, alanine aminotransferase (ALT), alkaline phosphatase (ALP)/serum glutamic pyruvic transaminase (SGPT), total bilirubin, creatinine, sodium, aspartate aminotransferase (AST)/serum glutamic oxaloacetic (SGOT), total protein, fasting blood glucose, calcium, carbon dioxide, estimated glomerular filtration rate (eGFR), and chloride
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Chemistry Parameters | 0 |
Urine samples were collected for the analysis of urinalysis parameters: Specific gravity, color, clarity, pH, glucose, protein, blood, nitrite, protein, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick, and microscopic examination (if blood or protein was abnormal)
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Number of Participants With Clinically Significant Changes in Urinalysis | 0 |
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. The score ranges from 1 to 5 and higher scores indicate worse symptoms.
| Participants | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Suicidal Behavior: Preparatory acts or behavior | 0 |
| Suicidal Behavior: Aborted attempt | 0 |
| Suicidal Behavior: Interrupted attempt | 0 |
| Suicidal Behavior: Actual attempt | 0 |
| Suicidal Behavior: Completed suicide | 0 |
| Suicidal Ideation: Wish to be dead | 0 |
| Suicidal Ideation: Non-specific Active thoughts | 0 |
| Suicidal Ideation: Active ideation without intention to act | 0 |
| Suicidal Ideation: Active ideation with some intent to act without a plan | 0 |
| Suicidal Ideation: Active ideation with a specific plan and intent | 0 |
| Non-suicidal Self-Injurious Behavior | 1 |
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28. Percent change from baseline in 28-day frequency was calculated for each participant by subtracting Baseline 28-day seizure frequency from post-Baseline 28-day seizure frequency, whole divided by Baseline 28-day seizure frequency and multiplied by 100.
| percent change | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Percent Change From Baseline in 28-day Seizure Frequency During Open Label Extension | -31.59 ± 113.997 |
Collected over Up to 150 Weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) | 1/117 (0.9%) | 21/117 (17.9%) | 79/117 (67.5%) |
| Event | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| SeizureNervous system disorders | 5/117 |
| PneumoniaInfections and infestations | 3/117 |
| GastroenteritisInfections and infestations | 2/117 |
| Atypical PneumoniaInfections and infestations | 1/117 |
| BronchitisInfections and infestations | 1/117 |
| Covid-19Infections and infestations | 1/117 |
| LaryngitisInfections and infestations | 1/117 |
| Pneumonia BacterialInfections and infestations | 1/117 |
| Pneumonia MycoplasmalInfections and infestations | 1/117 |
| Respiratory Syncytial Virus InfectionInfections and infestations | 1/117 |
| Event | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| SeizureNervous system disorders | 17/117 |
| Upper Respiratory Tract InfectionInfections and infestations | 10/117 |
| SomnolenceNervous system disorders | 10/117 |
| PyrexiaGeneral disorders | 9/117 |
| NasopharyngitisInfections and infestations | 7/117 |
| VomitingGastrointestinal disorders | 7/117 |
| PneumoniaInfections and infestations | 6/117 |
| FallInjury, poisoning and procedural complications | 6/117 |
| FatigueGeneral disorders | 5/117 |
| Covid-19Infections and infestations | 4/117 |
Safety analysis set comprised of all participants who received at least 1 dose of the investigational product (IP).
| Age, Continuous(years) | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Mean | 16.4 ± 11.2 |
| Sex: Female, Male(Participants) | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Female | 62 |
| Male | 55 |
| Ethnicity (NIH/OMB)(Participants) | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| Hispanic or Latino | 16 |
| Not Hispanic or Latino | 100 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Ganaxolone (GNX) Oral Suspension, 3 Times a Day (TID) |
|---|---|
| American Indian or Alaska Native | 1 |
| Asian | 24 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 8 |
| White | 82 |
| More than one race | 2 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Ganaxolone