Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment
This 'roll-over' study looks at the long-term safety of a medication called everolimus for people aged 2 to 65 with Tuberous Sclerosis Complex (TSC) and seizures that are hard to control. It's for patients who are currently taking everolimus in another study, EXIST-3, and whose doctors believe they are still benefiting from the treatment. The main goal is to keep collecting information on how safe everolimus is over a longer period. Patients will continue to receive everolimus as long as they are benefiting and meet the study's requirements. This research helps understand the medication better over time.
At a glance
What is this study about?
This study, called CRAD001M2304, is designed for individuals who have Tuberous Sclerosis Complex (TSC), a genetic condition that can cause non-cancerous growths in various organs, including the brain, leading to seizures. Specifically, this study focuses on patients whose seizures are 'refractory,' meaning they are difficult to control with standard treatments. The medication being studied is everolimus.
This isn't a new treatment study; instead, it's a 'roll-over' study. This means it's for patients who are already taking everolimus as part of another related study called EXIST-3. If their doctor believes they are still getting a benefit from everolimus treatment once the EXIST-3 study finishes, they might be invited to join this long-term safety study. The main purpose is to continue gathering important information about the safety of everolimus when used over many years.
The study is planned to run for a long time – up to 10 years in some cases, or until the end of 2022 in the UK. Patients will continue taking everolimus in this study until their doctor decides it's no longer helping, or if certain other conditions for stopping treatment are met. It's important to understand how treatments work and feel over an extended period, so this study is vital for learning more about everolimus.
Key takeaways
- This study is for long-term safety of everolimus.
- It's only for patients currently in the EXIST-3 study.
- You must be benefiting from everolimus treatment to join.
- The study could last for many years (up to 10).
- Regular health checks will be part of your participation.
- You can stop participating at any time you wish.
Who may be eligible?
To be considered for this study, you must already be taking part in the EXIST-3 study and receiving everolimus. Your doctor needs to confirm that you are currently benefiting from everolimus treatment and that you have consistently followed the rules of the EXIST-3 study so far. You also need to be willing and able to attend all scheduled appointments and follow the treatment plan.
Before joining, you (or your parent/guardian, if you are under 16) will need to give your written consent to take part after understanding all the details. For young people, adolescents may also need to give their assent, which means they agree to participate after hearing about the study.
However, you cannot join if you've already stopped taking everolimus in the EXIST-3 study. Also, if everolimus is already available and covered by the NHS or other local health services in your country for TSC and difficult-to-control seizures, you would not be eligible. Lastly, if you are combining everolimus with unapproved or experimental seizure treatments (though standard anti-epilepsy drugs are allowed), you wouldn't be able to join.
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 currently taking everolimus in the EXIST-3 study?
- Does your doctor believe everolimus is still helping you?
- Are you (or your child) aged between 2 and 65?
- Are you able to attend clinic visits regularly?
- Have you not previously stopped everolimus in the EXIST-3 study?
- Is everolimus not yet routinely available through the NHS for your condition?
What does participation involve?
If you are eligible and choose to take part, you will continue to receive everolimus, as you are currently doing in the EXIST-3 study. The study team will explain everything to you in detail. You will need to attend scheduled visits to the clinic, which will include check-ups, assessments, and doctors evaluating how you are doing. The exact number and frequency of visits will be explained by the study team, but they are crucial for monitoring your health and the effects of the medication.
The study is designed to run for a long time, potentially up to 10 years, or until the end of 2022 in the UK. During this time, you will continue to take everolimus. You'll remain in the study as long as your doctor believes you are still benefiting from the treatment, and you meet the study's ongoing requirements. If at any point your doctor feels the treatment isn't helping anymore, or if other specific reasons arise, your participation might end. You will always have the right to withdraw from the study at any time.
Potential risks and benefits
Locations (68)
- TGen APNNAVerified postcodePhoenix, United States
- David Geffen School of Medicine at UCLAVerified postcodeLos Angeles, United States
- UCSF Benioff Children s HospitalVerified postcodeOakland, United States
- Rady Children s HospitalVerified postcodeSan Diego, United States
- University of Colorado School of MedicineVerified postcodeAurora, United States
- Connecticut Childrens Medical CntrVerified postcodeHartford, United States
- University of Chicago Medical CenterVerified postcodeChicago, United States
- Minnesota Epilepsy GroupVerified postcodeSaint Paul, United States
- Atlantic Health SystemsVerified postcodeMorristown, United States
- Cinn Children Hosp Medical CenterVerified postcodeCincinnati, United States
- Oregon Health Sciences UniversityVerified postcodePortland, United States
- Childrens Hospital of PhiladelphiaVerified postcodePhiladelphia, United States
Common questions
What is a 'roll-over' study?
It's a study for patients who are already taking a treatment in another research study and are continuing to benefit, allowing them to carry on receiving the treatment and be monitored for a longer time.
What is the medication being studied?
The medication is called everolimus. It is being studied for its long-term safety in people with Tuberous Sclerosis Complex and difficult-to-control seizures.
How long would I be in the study?
The study aims to collect information for an extensive period, potentially up to 10 years or until the end of 2022 in the UK, as long as you are benefiting from the treatment.
Can I stop participating at any time?
Yes, absolutely. You are free to withdraw from the study at any point, for any reason, without affecting your medical care.
What happens after the study ends?
The study will end when your doctor feels the treatment is no longer helping, or if other specific conditions are met. You won't automatically receive everolimus; your doctor will discuss your future treatment options with you.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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