TReating Uncontrolled Seizures in Tuberous Sclerosis Complex
Are you or someone you care for diagnosed with tuberous sclerosis complex (TSC) and suffering from inadequate seizure control?
If you or your child have been diagnosed with TSC and have inadequate response to your existing regimen for seizure control, there may be a clinical study for you.
The TrustTSC study is exploring whether treatment with an investigational medication (ganaxolone) has the potential to reduce the frequency of seizures associated with TSC in children and adults.
Currently, there are a limited number of treatments approved specifically for individuals living with tuberous sclerosis complex (TSC). Anti-seizure medications that are available to treat seizures associated with TSC may not work for everyone, may stop working over time, or may have to be stopped because of side effects.
About TSC
Tuberous sclerosis complex (TSC) is a rare genetic disorder that is most likely not inherited, but can be passed on from parents to their children. With more than 50,000 people in the U.S. and approximately one million people worldwide living with TSC, the disorder may affect each person differently, with signs and symptoms ranging from very mild to severe. Because TSC affects everyone differently, there is no way to predict where and how the disorder will manifest.
TSC can affect different organs in the body causing tumors to develop in the brain, skin, kidney, heart, eyes, and lungs. Seizures are the most common neurological symptom of TSC and not all individuals with TSC have the same types of seizures—so one medication may not work for all. Gaining seizure control is one of the most critical priorities for any individual with TSC-associated seizures and all efforts should be made to reduce the total number of daily seizures.
Approximately 85% of TSC patients have epilepsy and approximately 2/3 of those patients are not adequately controlled with existing anti-seizure medications. Any therapy that reduces the frequency, duration or severity of seizures may positively impact quality of life for those individuals and their families.
What is the TrustTSC Study?
The TrustTSC study is exploring whether treatment with investigational ganaxolone can reduce the frequency of seizures associated with TSC in children and adults.
Ganaxolone is a neuroactive steroid that binds to GABAA receptors in the brain and is thought to help normalize over-excited neurons and provide a stabilizing effect. The purpose of the TrustTSC study is to determine whether ganaxolone could be effective as an adjunctive therapy to treat seizures in children and adults living with TSC.
The TrustTSC study is a global Phase 3 randomized, double-blind, placebo-controlled study of ganaxolone treatment in children and adults with TSC. The study consists of a 4-week baseline period defined as the first 28 days following screening (to collect seizure data), followed by a 16-week double-blind treatment phase, followed by either a 2-week down titration period or a 4-week crossover period for qualifying individuals who choose to enter the long term open-label phase.
At the screening visit to determine eligibility per inclusion/exclusion criteria, you will be asked about your seizure history, including seizure types, frequency, and seizure free days. After meeting the eligibility criteria and completing the 4-week baseline period, participants will be randomly assigned to receive either ganaxolone or placebo. Neither you, nor the study staff will know which study medication you are receiving. Participants must remain on the anti-seizure medications they are currently taking.
Do I Qualify?
Children and adults must meet the following
criteria to be eligible for the study:
Clinical or mutational TSC diagnosis (TSC1 or TSC2 mutation confirmed by your doctor)
2-65 years old
Inadequate seizure control
Treated with at least two prior antiseizure medications
Not currently enrolled in any other clinical studies
The study doctor will require a pre-screening for potential participants and will ask questions about medical history and seizures to determine eligibility. 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.
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 patient and caregiver.
Participation in the TrustTSC study is completely voluntary. Ask your doctor if being in this study is right for you or your child.
Clinical Research
Clinical research studies evaluate investigational medications to determine if they are safe and effective for patients before they are made available to the public. Studies are carefully designed and monitored by health professionals, with participant safety as a priority.
Before enrolling in clinical research studies, participants review and sign an informed consent document, stating that the study details have been explained to them and their questions have been answered. This document is not a contract; participation in a clinical research study is completely voluntary and study participants can leave the study for any reason at any time.
Frequently Asked Questions
Can participants take other antiseizure medications (ASMs) and participate in this study?
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.
Is there help for transportation costs?
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.
Can participants be seen by their regular physician?
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.
Am I guaranteed to get ganaxolone in this 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.
Who can join this study?
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.
What do I have to do to be considered for the study?
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.
What study treatment options are provided?
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.
Are there side effects?
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.
Will this help me/ my child?
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.
What is a clinical study?
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.