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Ganaxolone for Tuberous Sclerosis-Related Epilepsy (TrustTSC Trial)

Phase 3
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
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) and day 29 to week 16
Awards & highlights

TrustTSC Trial Summary

This trial is testing a possible new treatment for epilepsy in people with tuberous sclerosis complex. It is a Phase 3 study, which means it is one of the last stages of testing before the drug can be approved.

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.Check my eligibility
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.See study design
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.

TrustTSC Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
I have been diagnosed with TSC through genetic testing or clinical assessment.
I agree to use effective birth control methods.
I've had at least 8 seizures a month in the last 2 months.
My seizures have not stopped despite trying 2 or more medications.

TrustTSC Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) and day 29 to week 16
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) and day 29 to week 16 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Double-blind phase: Percent change from Baseline in 28-day seizure frequency during titration and maintenance period
Secondary outcome measures
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
+13 more

TrustTSC 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

Find a Location

Who is running the clinical trial?

Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,715 Total Patients Enrolled
2 Trials studying Tuberous Sclerosis
155 Patients Enrolled for Tuberous Sclerosis

Media Library

Ganaxalone (GNX) Clinical Trial Eligibility Overview. Trial Name: NCT05323734 — Phase 3
Tuberous Sclerosis Research Study Groups: Placebo matching GNX, Ganaxalone (GNX)
Tuberous Sclerosis Clinical Trial 2023: Ganaxalone (GNX) Highlights & Side Effects. Trial Name: NCT05323734 — Phase 3
Ganaxalone (GNX) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05323734 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this clinical trial target a specific age group?

"According to the specific guidelines set out by the trial, applicants who are 1 year to 65 years old are eligible. Out of the 656 total trials, 142 are for patients under 18 and 514 are for patients over 65."

Answered by AI

Are participants being currently accepted for this research?

"The clinicaltrials.gov website lists this trial as recruiting, with an original posting date of April 1st, 2022 and a most recent update of October 28th, 2022."

Answered by AI

How many guinea pigs are being used for this medical experiment?

"One hundred and sixty-two patients that fulfill the requirements set by the sponsor, Marinus Pharmaceuticals, are necessary to carry out this study. The trial will take place in various locations, including University of Florida Gainesville in Gainesville, Florida, and Children's Hospital of Orange County in Orange, California."

Answered by AI

Has the FDA cleared GNX oral suspension for use three times a day?

"GNX oral suspension, TID has some clinical evidence supporting its efficacy and multiple studies backing its safety, so it received a score of 3."

Answered by AI

Could you provide information about how many research facilities are participating in this study?

"To make things more convenient for potential participants, 67 sites are currently enrolling patients for this study. While the locations in Gainesville, Orange and Hershey are the closest, there are 67 other locations across the country."

Answered by AI

Who else is applying?

What site did they apply to?
Phoenix Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
~42 spots leftby May 2025