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Ganaxolone for TSC-Related Epilepsy
Phase 3
Waitlist Available
Research Sponsored by Marinus Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) and week 1 through week 156
Awards & highlights
Study Summary
This trial studies an adjunctive treatment in children & adults with Tuberous Sclerosis Complex (TSC) who participated in previous studies.
Who is the study for?
This trial is for children and adults with TSC-related epilepsy who have been part of previous studies (1042-TSC-3001 or 1042-TSC-2001). Participants must be able to maintain a seizure diary, take the study drug with food three times daily, use effective contraception if applicable, and not be pregnant or breastfeeding. They should not have other health conditions that could affect the study.Check my eligibility
What is being tested?
The trial is testing Ganaxolone as an additional treatment in those with Tuberous Sclerosis Complex (TSC) related epilepsy. It's an open-label extension meaning everyone knows they're getting Ganaxolone, which was previously studied in two earlier trials.See study design
What are the potential side effects?
While specific side effects for this trial are not listed, Ganaxolone may cause dizziness, tiredness, headache and potentially impact hormonal balance due to its nature as a steroid compound.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline (day 1) and week 1 through week 156
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) and week 1 through week 156
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of participants with abnormal 12-lead electrocardiogram (ECG) findings
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Number of participants with abnormal clinical laboratory tests
+3 moreSecondary outcome measures
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
Change from Baseline in percentage of Seizure-Free Days during treatment, based on seizure type
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
+4 moreSide effects data
From 2014 Phase 2 trial • 112 Patients • NCT0133968919%
Somnolence
14%
Headache
8%
Fatigue
8%
Sedation
7%
Dizziness
7%
Nightmare
7%
Vision blurred
7%
Myalgia
5%
Confusional state
5%
Dysarthria
3%
Vertigo
3%
Pain in extremity
3%
Gastrointestinal disorder
3%
Tremor
3%
Neck pain
3%
Nasopharyngitis
3%
Increased appetite
3%
Ataxia
3%
Balance disorder
3%
Cough
3%
Insomnia
3%
Diarrhoea
3%
Lethargy
2%
Road traffic accident
2%
Pulmonary congestion
2%
Splenomegaly
2%
Upper respiratory tract infection
2%
Dry mouth
2%
Confusion
2%
Arthralgia
2%
Balance Issues
2%
Tandem galt test abnormal
2%
Rhinorrhoea
2%
Urinary tract infection
2%
Protein urine present
2%
Vomiting
2%
Chest pain
2%
Decreased appetite
2%
Abnormal dreams
2%
Nausea
2%
Chest discomfort
2%
Feeling drunk
2%
Irritability
2%
Pharyngitis streptococcal
2%
Blood urine present
2%
Weight increased
2%
Back pain
2%
Muscle spasms
2%
Depressed level of consciousness
2%
Disturbance in attention
2%
Motor dysfunction
2%
Anger
2%
Depressed
2%
Euphoric mood
2%
Initial insomnia
2%
Psychomotor retardation
2%
Sleep disorder
2%
Upper airway resistance syndrome
2%
Anorgasmia
2%
Suicidal ideation
2%
Haematuria
2%
Suicide of companion
2%
Product used for unknown indication
2%
Dry Eye
2%
Abdominal discomfort
2%
Alanine aminotransferase increased
2%
Liver function test abnormal
2%
Tic
2%
Migraine
2%
Suicidal Ideation
2%
Fever
2%
Sinus congestion
2%
Rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ganaxolone
Placebo
Trial Design
1Treatment groups
Experimental Treatment
Group I: Ganaxolone (GNX) oral suspension, 3 times a day (TID)Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxolone
2011
Completed Phase 2
~680
Find a Location
Who is running the clinical trial?
Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,831 Total Patients Enrolled
2 Trials studying Tuberous Sclerosis
151 Patients Enrolled for Tuberous Sclerosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I agree to use effective birth control during and for 30 days after the study.I haven't used any experimental drugs or devices recently.I don't have any health issues that could affect how a drug works in my body.I do not have an active brain infection, multiple sclerosis, or a degenerative brain disease.
Research Study Groups:
This trial has the following groups:- Group 1: Ganaxolone (GNX) oral suspension, 3 times a day (TID)
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is GNX a viable option for patients with few other options?
"There is some evidence of GNX's efficacy and multiple reports of its safety, so it was given a score of 3."
Answered by AI
Who would the doctors consider to be an ideal candidate for this research?
"This study seeks out 169 individuals that are between the ages of 1 year to 65 years old, have aura, and meet the following requirements: Being able to complete Study 1042-TSC-3001 or already meeting the study requirements in Study 1042-TSC-2001, being willing to give written informed consent/assent after being properly informed of the study's procedures and risks, being able to maintain an accurate and complete daily seizure diary, being able to take IP suspension with food three times a day, being on a medically acceptable birth control method if sexually active and female with a negative quantitative serum β-HCG"
Answered by AI
Are younger individuals eligible for this research project?
"In order to take part in this experiment, potential patients must between the ages of 1 and 65 years old."
Answered by AI
Is this study currently open to new participants?
"The clinical trial mentioned is not currently enrolling patients, as per the latest update on clinicaltrials.gov. This particular trial was posted on May 16th, 2022 and last updated on November 2nd, 2022. There are, however, 741 other trials that are currently looking for patients."
Answered by AI
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