Ganaxolone for Tuberous Sclerosis-Related Epilepsy

(TrustTSC Trial)

Recruiting at 129 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Marinus Pharmaceuticals

Stay on Your Current Meds

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

Participant/parent(s) or LAR(s) willing to give written informed consent/assent

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.

Exclusion Criteria

Participants deprived of liberty or admitted to a facility for purposes other than research

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

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

Treatment Details

Interventions

• Ganaxalone (Corticosteroid)
• GNX oral suspension (Other)
• Placebo (Other)

Trial Overview 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.

Participant Groups

Group I: Ganaxalone (GNX) Experimental Treatment 1 Intervention

oral suspension, 3 times a day (TID)

Group II: Placebo matching GNX Placebo Group 1 Intervention

oral suspension, TID

Who Is Running the Clinical Trial?

Marinus Pharmaceuticals

Lead Sponsor

Trials

23

Recruited

1,800+