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Ganaxolone for Tuberous Sclerosis-Related Epilepsy (TrustTSC Trial)
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
Clinical or mutational diagnosis of TSC consistent with molecular confirmation of a pathogenic mutation in TSC1 or TSC2, or clinical diagnosis of definite TSC
Male participants must agree to use highly effective contraceptive methods
Must not have
Epilepsy surgery planned during the study or surgery within the past 6 months
Concurrent use of ACTH, prednisone, or strong CYP3A4 inducers
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) through week 16
Awards & highlights
Pivotal Trial
Summary
This trial is testing a medication called GNX to see if it can help reduce seizures in children and adults with epilepsy caused by Tuberous Sclerosis Complex (TSC). The medication works by calming overactive brain signals. The study includes a time to adjust the dosage and a time to see if the treatment is effective.
Who is the study for?
This trial is for children and adults aged 1-65 with TSC-related epilepsy who have frequent seizures despite trying at least two anti-seizure medications. They must not be planning epilepsy surgery soon, should not have had recent exposure to the study drug or certain other treatments, and women of childbearing age must use birth control.
What is being tested?
The trial tests Ganaxolone (GNX), an adjunctive treatment for epilepsy related to Tuberous Sclerosis Complex (TSC). Participants will either receive GNX or a placebo over a period that includes titration (4 weeks) and maintenance phases (12 weeks), in addition to their current seizure medications.
What are the potential side effects?
While specific side effects are not listed here, participants may experience adverse reactions related to Ganaxolone which could include dizziness, fatigue, headache, gastrointestinal disturbances or potential hormonal imbalances due to its steroid structure.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with TSC through genetic testing or clinical assessment.
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I agree to use effective birth control methods.
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I've had at least 8 seizures a month in the last 2 months.
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My seizures have not stopped despite trying 2 or more medications.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am scheduled for epilepsy surgery or had surgery in the last 6 months.
Select...
I am currently taking ACTH, prednisone, or strong CYP3A4 inducers.
Select...
I do not have an active brain infection or a disease causing brain deterioration.
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I have been treated with GNX before.
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I have liver, bile duct, or kidney problems.
Select...
I am using CBD products that are not Epidiolex.
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My drug screen for CBD or THC was positive.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline (day 1) through week 16
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) through week 16
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Double-blind phase: Percent change from Baseline in 28-day seizure frequency during titration and maintenance period
Secondary study objectives
Double-blind phase: Change from Baseline in Anxiety, Depression, and Mood Scale (ADAMS) during titration and maintenance phase
Double-blind phase: Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID) during titration and maintenance phase
Double-blind phase: Change from Baseline in Epilepsy and Learning Disabilities Quality of Life (ELDQOL) Scale during titration and maintenance phase
+4 moreAwards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Ganaxalone (GNX)Experimental Treatment1 Intervention
oral suspension, 3 times a day (TID)
Group II: Placebo matching GNXPlacebo Group1 Intervention
oral suspension, TID
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxalone
2022
Completed Phase 3
~130
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Tuberous Sclerosis (TSC) often target the mTOR pathway, which is crucial in regulating cell growth and proliferation. Medications like everolimus inhibit mTOR, helping to reduce the size and number of tumors associated with TSC.
Ganaxolone, a GABA-A receptor modulator, works by enhancing the inhibitory effects of GABA in the brain, which can help manage seizures—a common symptom in TSC patients. These treatments are significant because they address both the growth of benign tumors and the neurological symptoms, improving the overall quality of life for TSC patients.
Find a Location
Who is running the clinical trial?
Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,645 Total Patients Enrolled
2 Trials studying Tuberous Sclerosis
155 Patients Enrolled for Tuberous Sclerosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am scheduled for epilepsy surgery or had surgery in the last 6 months.I am currently taking ACTH, prednisone, or strong CYP3A4 inducers.I have been diagnosed with TSC through genetic testing or clinical assessment.I have been on a stable epilepsy medication regimen for at least 28 days.I have not used traditional Chinese medicine in the last 28 days.I agree to use effective birth control methods.I do not have an active brain infection or a disease causing brain deterioration.I am between 1 and 65 years old.I've had at least 8 seizures a month in the last 2 months.I can take the study drug with food as instructed.I have been treated with GNX before.I have been taking felbamate for less than a year.I have liver, bile duct, or kidney problems.I am using CBD products that are not Epidiolex.My drug screen for CBD or THC was positive.I haven't changed my long-term medications in the last 4 weeks.I don't have any health issues that could risk my safety or affect the study's results.My seizures have not stopped despite trying 2 or more medications.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo matching GNX
- Group 2: Ganaxalone (GNX)
Awards:
This trial has 1 awards, including:- 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.