The purpose of the study is to evaluate how effective and safe the treatment is in reducing the number of epileptic seizures in patients with refractory epilepsy due to TSC. Participants will be given either the YCJ-01 treatment or a placebo over a period of time. The study will monitor changes in the frequency of seizures and any side effects that may occur. The treatment is administered orally, meaning it is taken by mouth in the form of an oil.

Throughout the study, participants will have regular check-ups to assess their response to the treatment. The trial aims to determine if the YCJ-01 treatment can significantly decrease the number of seizures compared to the baseline period before the treatment started. The study will also compare the results between those receiving the actual treatment and those receiving the placebo to understand the treatment’s effectiveness better. The trial is expected to continue until the end of 2026.

Participants in the study will be randomly assigned to receive either Rapamune, Sabril, or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment. This helps ensure that the results are not biased. The trial will last for a period of up to two years, during which the infants’ health will be closely monitored. Researchers will look at various factors, such as the occurrence of seizures, the growth of TSC-related tumors, and the overall development of the infants.

Throughout the study, the infants will undergo regular check-ups to monitor their physical development, including weight and height, as well as vital signs like body temperature and blood pressure. The study will also assess the risk of developing conditions such as autism and drug-resistant epilepsy. By the end of the study, researchers hope to gather valuable information on the effectiveness and safety of Rapamune and Sabril in preventing the symptoms of TSC in infants, which could lead to better treatment options in the future.

The purpose of this study is to evaluate the safety and effectiveness of rapamycin compared to a placebo in reducing seizures in patients with drug-resistant epilepsy linked to TSC. Participants in the study will be randomly assigned to receive either rapamycin or a placebo. The study will monitor the number of seizures participants experience and any side effects that may occur. The trial will last for a period of time, during which participants will be closely observed to gather information on how well the treatment works and how safe it is.

Throughout the study, researchers will compare the outcomes between those receiving rapamycin and those receiving the placebo. The main goal is to determine if rapamycin can significantly reduce the frequency of seizures and improve the quality of life for individuals with TSC-related epilepsy. The study will also track any adverse events, which are unwanted effects that might occur during the treatment period. This information will help in understanding the potential benefits and risks of using rapamycin for this condition.

The purpose of the study is to assess how well Basimglurant works in reducing seizures when used together with current seizure medications. Participants in the study will be randomly assigned to receive either Basimglurant or a placebo, which looks like the medication but does not contain the active ingredient. The study will be conducted over a period of 30 weeks, during which participants will not know whether they are receiving Basimglurant or the placebo. After this period, there will be an additional 52-week phase where all participants will receive Basimglurant.

Throughout the study, participants will continue their regular anticonvulsive therapy. The study will monitor the number of seizures, any changes in daily activities, and overall impressions of change from caregivers. Safety will also be closely observed, including any side effects or changes in health. This trial is designed to provide valuable information on whether Basimglurant can be a beneficial addition to existing treatments for those with Tuberous Sclerosis Complex.

The purpose of this study is to gather information on the long-term safety of everolimus in patients who have already participated in a previous study called EXIST-3. Patients in this trial are those who have been determined by their doctors to benefit from continuing treatment with everolimus. The study is designed to monitor the safety of the medication over an extended period, ensuring that it remains a viable option for managing symptoms associated with TSC.

Participants in the study will continue to take everolimus as prescribed by their healthcare provider. They will attend regular visits with their doctor to assess their condition and any potential side effects. The study aims to provide valuable data on the frequency and severity of any adverse effects experienced by patients, as well as the overall clinical benefits observed during the treatment period.

Participants in this study will be between the ages of 1 and 65. The study will involve taking Epidyolex as an additional therapy to their existing treatment. The study will last for a period of up to 56 weeks, during which participants will receive the medication and be monitored for changes in their behavior and seizure activity. The study will also look at how the medication impacts the quality of life and family functioning, as well as any changes in sleep patterns and overall symptom severity.

Throughout the study, participants will have regular check-ins to assess their progress and any side effects they may experience. The study aims to provide valuable insights into how Epidyolex can help manage the symptoms of TSC, particularly focusing on the most challenging behaviors identified by caregivers. This research could potentially lead to improved treatment options for individuals living with TSC and their families.

Participants in the study will receive either the sirolimus treatment or a placebo, in addition to the standard care they would normally receive. The study will monitor the children’s development over a period of 24 months, assessing their cognitive abilities using a tool called the Bayley Scales of Infant and Toddler Development III (BSID-III). This will help determine if the treatment has a positive impact on their neuropsychological development compared to standard care alone.

Throughout the study, various aspects of the children’s health will be closely monitored, including their behavior, signs of autism spectrum disorder, and any changes in seizure frequency. The study will also look at the size and number of tumors in the brain and heart, as well as any potential side effects of the treatment. The goal is to gather comprehensive data on the safety and effectiveness of sirolimus in improving the quality of life for children with tuberous sclerosis complex.

The purpose of the study is to evaluate the safety and effectiveness of this treatment over a period of 52 weeks. Participants will receive the Cannabidiol oral solution in addition to their current seizure medications. The study will monitor how the body processes the medication and its effects on seizure frequency. The trial is open-label, meaning all participants will receive the active treatment, and there is no placebo group. The study will involve regular check-ups to assess the health and development of the participants, as well as to monitor any side effects or changes in seizure patterns.

Throughout the study, caregivers will be asked to keep a diary of the participant’s seizures to help researchers understand how well the treatment is working. The study aims to provide valuable information on whether Cannabidiol can be a safe and effective addition to existing treatments for these challenging epilepsy conditions in young children.

The purpose of the study is to assess the safety and tolerability of Radiprodil, as well as how the body processes the medication. Participants will receive multiple doses of Radiprodil that are adjusted individually. The study will also look at how the medication affects seizure frequency and behavioral symptoms over time. Participants will be monitored for any side effects, changes in vital signs, and other health indicators throughout the study.

The study is open-label, meaning both the participants and the researchers know which treatment is being administered. It will involve regular check-ups and assessments to track the effects of Radiprodil on the participants’ conditions. The study will also use tools like electronic diaries to record seizure activity and other symptoms. The goal is to gather comprehensive data on how Radiprodil can benefit patients with these specific neurological conditions.