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Long-Term Safety Study of Everolimus for Patients with Tuberous Sclerosis Complex and Refractory Seizures

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on patients with tuberous sclerosis complex (TSC), a genetic disorder that causes non-cancerous tumors to form in many parts of the body, and who experience refractory seizures, which are seizures that do not respond well to standard treatments. The study involves the medication everolimus, which is taken as a dispersible tablet. Everolimus is a chemical substance that has been used in previous studies to help manage symptoms in patients with TSC.

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment in the roll-over study

    Enrollment in the roll-over study occurs after completion of the EXIST-3 study. Participation requires current enrollment in the EXIST-3 study, receiving everolimus, and fulfilling all its requirements.

    The patient must be benefiting from everolimus treatment, as determined by the investigator, and must have demonstrated compliance with the parent study protocol requirements.

    Written informed consent or adolescent assent is required prior to enrolling in the roll-over study.

  2. Step 2

    Treatment with everolimus

    The patient continues treatment with everolimus, administered orally. The dosage and frequency are determined by the investigator based on individual needs and response to treatment.

    The treatment aims to manage refractory seizures associated with tuberous sclerosis complex (TSC).

  3. Step 3

    Scheduled visits and assessments

    Regular visits are scheduled to monitor the patient's response to everolimus and to assess the frequency and severity of any adverse events (AEs) or serious adverse events (SAEs).

    The investigator evaluates the clinical benefit of the treatment at these visits.

  4. Step 4

    Long-term safety evaluation

    The main objective of the study is to evaluate long-term safety data of everolimus in patients with TSC and refractory seizures.

    The study is expected to continue until August 30, 2027, providing ongoing assessment of safety and clinical benefit.

Who can join the trial?

5 criteria

  • The patient must be currently enrolled in the Novartis-sponsored EXIST-3 study and receiving a medication called **everolimus**.
  • The patient must be benefiting from the treatment with **everolimus**, as determined by the study doctor.
  • The patient must have followed the rules and requirements of the previous study, as checked by the study doctor.
  • The patient must be willing and able to attend scheduled visits and follow the treatment plans.
  • The patient or their guardian must provide written permission to join the new study. For teenagers, an agreement called **adolescent assent** is also needed.

Who cannot join the trial?

4 criteria

  • Patients who do not have **refractory seizures** associated with **tuberous sclerosis complex**. **Refractory seizures** are seizures that do not respond well to treatment. **Tuberous sclerosis complex** is a genetic disorder that causes non-cancerous tumors to form in different parts of the body.
  • Patients who are not within the specified age range for the study. The study includes certain age groups, so patients must fall within these age categories to participate.
  • Patients who are not male or female, as the study includes both genders.
  • Patients who are not considered part of a **vulnerable population**. A **vulnerable population** includes groups of people who may be at a higher risk of harm or exploitation, such as children, pregnant women, or those with certain disabilities.
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Investigated drugs

Everolimus is a medication used in this clinical trial for patients with tuberous sclerosis complex (TSC) who experience seizures that are difficult to control with other treatments. The trial aims to assess the long-term safety of continuing treatment with everolimus for these patients. Everolimus works by targeting specific pathways in the body that are involved in cell growth and immune function, which can help reduce the frequency and severity of seizures in patients with TSC.

What is already known about the treatment

Everolimus – Everolimus is administered orally in the form of tablets. It is currently being studied in clinical trials for its long-term safety in treating refractory seizures associated with tuberous sclerosis complex. The medication is primarily used to manage these seizures, which do not respond well to other treatments. At the molecular level, everolimus works by inhibiting a protein called mTOR, which plays a role in cell growth and proliferation. It is classified as an mTOR inhibitor in pharmacology.

Investigated diseases

Tuberous Sclerosis Complex – Tuberous sclerosis complex is a genetic disorder that causes non-cancerous tumors to form in many different organs, primarily the brain, eyes, heart, kidney, skin, and lungs. The condition is characterized by a variety of symptoms, including skin abnormalities, seizures, and developmental delays. Seizures associated with this condition can be particularly challenging to manage and may not respond well to standard treatments, leading to what is known as refractory seizures. These seizures can vary in type and severity, often requiring specialized care. The progression of the disease can differ significantly from person to person, with some experiencing more severe symptoms than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516746-19-00Protocol codeCRAD001M2X02BEstimated enrolment177 patientsSponsorNovartis Pharma AG

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