Frequently asked questions

The potential new treatment is investigational radiprodil. It specifically targets the overactive areas of the outer, folded layer of the brain (cortex), which are involved in causing seizures in TSC and FCD Type II.

Investigational radiprodil has not been approved by your country’s drug approval office or regulatory agencies for use in children or adolescents with TSC or FCD.

Everyone in the ASTROSCAPE Study will take investigational radiprodil, as well as their usually prescribed medicines (including rescue/emergency medicines). Stable ketogenic diets are also allowed. There is no placebo (inactive medicine) in this study.

Study participants will take investigational radiprodil twice a day by mouth, or by gastrostomy tube (G-tube) or nasogastric tube (NG-tube) if appropriate. They will take investigational radiprodil for approximately 18 weeks in Part A of the study, with the potential to continue to take it for another 1 year if they enter Part B of the study.

In addition to taking investigational radiprodil, you will be asked to bring your child to the clinic for health checks. These include health questionnaires, physical examinations, blood samples, pregnancy tests (if applicable), electrocardiograms (ECGs) and video electroencephalograms (V-EEGs). These assessments help us to monitor your child’s safety and well-being. Some of these clinic visits may be overnight stays in hospital.

There will be about 13 clinic visits in Part A of the study and about 6 clinic visits in Part B. Telephone calls will be scheduled in between clinic visits to check on your child’s safety and well-being.

Study participants or their caregivers will need to complete a daily electronic diary throughout the study to record the number and type of seizures. All medicines and the amount of investigational radiprodil administered will also need to be recorded.

How long your child will be in the study depends upon a few things, including how well they tolerate investigational radiprodil, how they respond to it, the study doctor’s assessment and personal choice.

The potential new treatment, investigational radiprodil, is a liquid to be given twice a day at home (or at the clinic on study visit days). The liquid may be given by mouth or by gastrostomy tube (G-tube) or nasogastric tube (NG-tube) if appropriate.

This test shows how your child’s heart is working. A technician will place small sticky pads on their chest, arms and legs that are connected by wires to an ECG machine. These pads collect a signal that measures the activity of your child’s heart.

A V-EEG records what your child is doing or experiencing on video while an EEG test records their brainwaves. The purpose is to be able to see what is happening when they have a seizure or event and compare the picture to what the EEG records at the same time. Sounds that occur during the testing are also recorded. This can pick up if your child talks or makes sounds during an event. By doing this, doctors reading the EEG can tell how the seizure or event was related to the electrical activity in the brain.

Like all medicines and medical procedures, clinical research has some risk, which could include side effects that make your child feel unwell or uncomfortable. However, your child will be checked regularly throughout the study by the study team.

There is no guarantee that your child will benefit in any way. Their health may improve, worsen or stay the same. However, your child’s participation in this study may benefit patients with drug-resistant epilepsy in the future.

Clinical trials, also known as clinical research or scientific studies, are conducted by doctors and researchers to see if new drugs or treatments are safe and effective before being approved by the general public. For basic information about clinical trials, go to: https://www.fda.gov/patients/clinical-trials-what-patients-need-know/basics-about-clinical-trials.

Research studies test new medicines for safety, tolerability and effectiveness before they are approved for use by the public.

You can learn more about clinical trials by visiting www.clinicaltrials.gov. This is a government online database maintained by the National Library of Medicine. You will find information on both government and privately funded clinical trials.

For all scientific studies, physicians and/or researchers must provide interested participants with complete and accurate information about the risks, benefits, and activities of a scientific study. Informed consent means that you and your child are provided with sufficient information to enable an informed decision about participation in the ASTROSCAPE Study. During the process, you will have ample opportunity to ask questions and consider whether you would like to participate. This process takes place under conditions that minimize the possibility of coercion or undue influence.