What side effects are associated with this type of intervention?

Common treatments for Tuberous Sclerosis Complex (TSC)-related epilepsy include antiepileptic drugs (AEDs) such as vigabatrin, everolimus, and cannabidiol. For treatments similar to Ganaxolone, which is a GABA-A receptor modulator, side effects can include drowsiness, dizziness, fatigue, and potential behavioral changes. Other AEDs may also cause side effects like nausea, weight gain, and potential liver enzyme elevation. It is important to monitor these side effects closely and discuss them with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Tuberous Sclerosis Complex (TSC), particularly for managing epilepsy associated with the condition. One notable approval is for Everolimus (marketed as Afinitor), which targets the mTOR pathway and is used to treat TSC-related seizures. While Ganaxolone, a GABA-A receptor modulator, is being studied for its efficacy in TSC-related epilepsy, other similar treatments include Vigabatrin (Sabril), which is approved for infantile spasms in TSC. These treatments aim to reduce seizure frequency and improve quality of life for patients with TSC.

What other types of research exist?

Promising novel alternatives to GNX for Tuberous Sclerosis patients include cannabidiol (CBD), which has shown efficacy in reducing seizure frequency in treatment-resistant epilepsy, including TSC-related epilepsy. Another alternative is everolimus, an mTOR inhibitor, which has been approved for treating TSC-related seizures and has demonstrated significant seizure reduction in clinical trials. Both treatments offer new avenues for managing TSC-related epilepsy and may be considered in consultation with a healthcare provider.

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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 below

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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:

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I am scheduled for epilepsy surgery or had surgery in the last 6 months.

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I am currently taking ACTH, prednisone, or strong CYP3A4 inducers.

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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.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) through week 16 for reporting.

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 more

Awards & 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

0 / 11Eligibility criteria met

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.