Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

To be considered for this study, participants need to be between 6 months and 18 years old and have a diagnosis of Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia Type II (FCD Type II). Importantly, their seizures must have been difficult to control, meaning they haven't responded well to at least two different seizure medicines.

Participants should also have had a certain number of seizures (at least 8 countable seizures over a recent four-week period) and a recent brain scan (MRI) within the last year. Any other treatments they are receiving for epilepsy or behaviour, such as special diets or devices, should have been stable for at least 28 days before joining the study.

However, some things would mean someone couldn't join. For example, if they have other significant medical problems not related to TSC or FCD Type II, or if they've had brain surgery within the last six months. Also, if they have certain serious liver problems or are taking specific medications that interact with radiprodil, they wouldn't be able to participate.

If you join this study, your involvement would typically start with a ‘Screening/Observation Period’ of up to six weeks. During this time, the study team will check if you're suitable and closely monitor your seizures before you start any new medicine.

Next comes the 'Titration Period' (about four weeks). You'll start taking radiprodil twice a day, and the dose will be slowly increased to find the safest and potentially most effective amount for you. The doctors will check how you're feeling and measure the medicine level in your blood.

After this, you'll enter the 'Maintenance Period' (about twelve weeks), where you'll continue taking that stable dose. Throughout these periods, you'll have regular hospital visits for check-ups and to see how you're responding. Your total participation could last around six months in Part A.

If the medicine is helping and is well-tolerated, you might be invited to join 'Part B,' which is a longer-term treatment phase. This could extend your participation to about one year or even longer. At the end of the study, if you stop taking the medicine, the dose will be gradually reduced over 15 days, followed by a final safety check-up two weeks later.