Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An open-label, multi-center long-term safety roll-over study in patients with tuberous sclerosis complex (TSC) and refractory seizures who are judged by the Investigator to benefit from continued treatment with everolimus after completion of Study CRAD001M2304 (EXIST-3)

This study is for adults (age 18 and over) who have a genetic condition called tuberous sclerosis complex, or TSC, and experience seizures that are hard to treat with regular medicines. Participants in this study have already taken part in a previous clinical trial for everolimus (known as EXIST-3) and their doctors believe the medication is helping them. The main purpose of this follow-up study is to continue monitoring the safety of everolimus over a longer period. Researchers will be carefully checking for any side effects and how often they occur, as well as their severity. They will also assess if patients continue to experience benefits from taking everolimus, as judged by their doctors. Both men and women can take part.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Novartis Pharma AG

Enrolment target

Start

18 Nov 2024

Refractory seizures associated with tuberous sclerosis complex

What is this study about?

Imagine you have a condition called tuberous sclerosis complex, or TSC. This is a genetic condition that can affect many parts of the body, including the brain, where it can cause seizures that are very difficult to control, even with several different medications.

Researchers are running a study called an 'open-label, multi-center long-term safety roll-over study'. Let's break that down. 'Open-label' means both you and your doctor will know you are taking the study medicine, everolimus. 'Multi-center' means the study is happening in many different hospitals or clinics, not just one. 'Long-term safety roll-over' means it's a way for people who have already taken part in a previous study (Study CRAD001M2304, also known as EXIST-3) to continue receiving everolimus if their doctor believes it's still helping them. The main goal is to keep a close eye on the safety of everolimus over a longer time.

The main thing researchers want to find out is how often side effects happen, how serious they are, and if new ones appear over time. They will also be asking your doctor to assess if you are still feeling better or benefiting from the treatment at your regular check-ups. This study is important because it helps scientists understand the long-term effects of everolimus for people with TSC who have hard-to-treat seizures, ensuring it's as safe and effective as possible for long-term use.

Key takeaways

This study is for adults with TSC and hard-to-control seizures.
Participants must have been in a previous everolimus study (EXIST-3).
The main goal is to monitor the long-term safety of everolimus.
Doctors will also assess if patients continue to benefit from the treatment.
Participation involves regular check-ups to monitor health and side effects.

Who may be eligible?

This study is specifically designed for adults aged 18 and older. Both men and women can participate. The most important requirement is that you must have tuberous sclerosis complex (TSC) and be experiencing seizures that are difficult to control, even with other treatments.

Crucially, you must have already taken part in a previous study investigating everolimus, known as Study CRAD001M2304 (EXIST-3). Your doctor must also believe that you are still getting a benefit from taking everolimus and that it's important for you to continue the treatment.

If you meet these specific criteria, you might be a good fit for this ongoing safety study. The study aims to gather more information about how everolimus affects people over a longer period.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have tuberous sclerosis complex (TSC)?
Do you experience seizures that are difficult to control?
Have you previously taken part in the EXIST-3 study that used everolimus?
Does your doctor believe that everolimus is still helping you?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would continue to take the medicine called everolimus, as you have been doing in the previous study. You would have regular visits to your study doctor or clinic. These visits will involve various assessments to monitor your health and how you're responding to the medication. This will include checking for any side effects (called 'adverse events' or 'serious adverse events' in medical terms) and their severity. Your doctor will also regularly assess whether they believe you are still benefiting from the treatment. The total duration of your participation will depend on the study design and your individual response, but it is focused on long-term follow-up.

Potential risks and benefits

The potential benefit of taking part is that you can continue to receive everolimus, a treatment that your doctor believes is helping to manage your difficult-to-control seizures caused by TSC. This could lead to continued improvement in your seizure control and overall well-being. However, like all medicines, everolimus can have side effects, which are potential risks. The study will carefully monitor these side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Poland

Common questions

What is everolimus?

Everolimus is a medication used to treat certain conditions, including seizures in people with tuberous sclerosis complex. It works by targeting specific pathways in the body.

Why is this study only for people who were in another study?

This study is a continuation of a previous study (EXIST-3). It allows researchers to gather more information on the long-term safety and effects of everolimus for people who have already been taking it and whose doctors believe it's working for them.

Will I know if I'm getting the real medicine?

Yes, this is an 'open-label' study, which means both you and your doctor will know that you are taking everolimus throughout the study.

What happens at the study visits?

At your study visits, the medical team will check your overall health, monitor for any side effects, and your doctor will assess how well the everolimus is managing your seizures. This helps ensure your safety and track the medication's effectiveness over time.

Can I stop taking part in the study if I want to?

Absolutely. You can choose to leave the study at any time, for any reason, without having to explain why, and it will not affect your usual medical care.