TReating Uncontrolled Seizures in Tuberous Sclerosis Complex

Yes. To be eligible, participants must have inadequate seizure control and been treated with at least two prior ASMs (although there are exceptions for younger participants). The TrustTSC study will allow enrollment of participants taking a full range of concomitant medications, including *EPIDIOLEX®/ EPIDYOLEX.

_{*EPIDIOLEX / EPIDYOLEX is a trademark of GW RESEARCH LIMITED.}

If you require financial assistance to get to a required study visit, the sponsor will provide travel concierge services or reimbursement of reasonable travel expenses for the participant and caregiver.

For this clinical study, participants will be seen by the physician that is conducting the study. However, participants should also continue to see their regular physician, who should be notified of your treatment and condition throughout the study.

The selection of who receives ganaxolone or placebo in the study treatment period is completely random with a 50% chance of receiving ganaxolone. During the treatment period, the study medication is blinded (unknown) to the study physician and staff, sponsor, participants, and caregivers. Following the 16-week double-blind treatment period, all completing participants have the choice to receive ganaxolone during the open-label phase of the study. During the open-label phase, all participants will receive ganaxolone.

Individuals from the ages of 1 to 65 who have a clinical or mutational diagnosis of TSC with inadequate seizure control and who have been treated with at least two prior ASMs may be eligible for the study. In addition, participants cannot be enrolled in another study with an investigational product in which they received treatment within 30 days prior to participating in TrustTSC.

See clinicaltrials.gov or EudraCT for details or ask your doctor if being in this study is right for you or your child.

Prior to enrolling in the study, participants may need to go through a pre-screening process where the study doctor will ask questions about medical history and seizure history to see if you are a good candidate for the study. You will be asked to keep track of you/ your child’s seizures (including the type and number of seizures every day, and the days when you/ your child doesn’t have any seizures) for four weeks.

Participants will be randomized into the clinical study to receive either ganaxolone or a placebo (non-active substance), in addition to their standard anti-seizure medication. The drug will be administered three times a day as a drinkable liquid, and it must be taken with food. All other medications must remain at a stable dosage while in the clinical study unless adjustments are deemed medically necessary by a physician.

All medicines can have side-effects. Ganaxolone has been generally well-tolerated in clinical studies to date. Over 1,900 people ranging from 4 months to 88 years of age have taken ganaxolone, some for as long as four years. Common side effects include sleepiness, dizziness, and fatigue.

An important aim of the TrustTSC study is to make sure that the health benefits of ganaxolone outweigh any risk or inconvenience from side effects. Before entering the study, participants will receive more detailed information on which side effects of ganaxolone treatment are already known from previous studies, and you are encouraged to talk about potential side effects with your study doctor.

Throughout the studies, your health and wellbeing will be closely monitored. If you have any specific concerns or think you might be experiencing a side-effect, you can talk to the study team at any time.

Previous studies have indicated that ganaxolone may have the ability to reduce seizures associated with TSC. This study intends to find out if ganaxolone is more effective in reducing seizures compared to placebo and whether there are other benefits associated with ganaxolone treatment in TSC patients. Marinus cannot promise any benefits to participants in this research study.

Half of the participants in the study will be in the control group that receives the placebo and will not receive investigational ganaxolone. Ask your doctor if being in this study is right for you.

Clinical studies are research studies used to help advance the understanding of a disease and find better ways to treat diseases, such as genetic epilepsies. Researchers use clinical studies to learn whether a potential drug is safe and effective.