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.
Long-term Safety Study for Tuberous Sclerosis Complex
Plain English Summary
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 is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Tuberous Sclerosis Complex. This study checks if everolimus is safe for long-term use in patients with TSC and seizures who have benefited from it. It's for patients who are currently in the EXIST-3 study and are getting better with everolimus. Participants take everolimus tablets and visit the doctor every 12 weeks for check-ups. Other treatments like AEDs can be used for seizures, but not unapproved or experimental ones. The trial aims to enroll 206 participants.
Official Summary
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be benefiting from continued treatment as judged by the investigator at the completion of EXIST-3
Who Can Participate
Here is what you need to know about eligibility for this trial. Patients must be in the EXIST-3 study and getting better with everolimus. They must have taken everolimus as required and be willing to keep taking it. The study is for adults and children with TSC and seizures who are benefiting from everolimus. Everolimus is not approved for all countries, so local coverage is important. This trial is studying Tuberous Sclerosis Complex, so participants generally need a confirmed diagnosis.
What They're Measuring
The study measures how often patients have side effects and how many see clinical benefits, helping patients know if the treatment is safe and effective over time. The specific primary outcome measures are: Occurances of adverse events and serious adverse events (Day 1 up to approximately 10 years). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.
Why This Trial Matters
This trial is crucial as it fills the gap in understanding the long-term safety of everolimus for TSC patients. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Tuberous Sclerosis Complex, where improved treatment options are needed.
Investor Insight
The large market size and Novartis' strong position make this a promising investment opportunity with a high approval probability. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.
Is This Trial Right for Me?
Ask your doctor if you are currently in the EXIST-3 study and getting better with everolimus. You will take everolimus tablets and visit the doctor every 12 weeks for check-ups. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NA
- Model: SINGLE_GROUP
- Masking: NONE
- Enrollment: 206 participants
Interventions
- DRUG: everolimus — everolimus, 2mg dispersible tablets
Primary Outcomes
- Occurances of adverse events and serious adverse events (Day 1 up to approximately 10 years)
Secondary Outcomes
- Percentage of patients with clinical benefit (Day 1 up to approximately 10 years, assessed every 12 weeks,)
Full Eligibility Criteria
Key Inclusion Criteria: * Patient is currently enrolled in the Novartis-sponsored EXIST-3 study, receiving everolimus, and has fulfilled all its requirements * Patient is currently benefiting from treatment with everolimus, as determined by the Investigator. * Patient has demonstrated compliance, as assessed by the Investigator,with the parent study protocol requirements. * Patient is willing and able to comply with scheduled visits and treatment plans. * Written informed consent/adolescent assent obtained prior to enrolling into the roll-over study. Key Exclusion Criteria: * Patient has been permanently discontinued from everolimus study treatment in EXIST-3 study * Everolimus is approved for patients with TSC and refractory seizures and is reimbursed in the local country. * Patients who are receiving everolimus in combination with unapproved or experimental treatments for seizure control Anti-epileptic drug (AEDs) are allowed for the purpose of seizure control.
Trial Locations
- TGen APNNA, Phoenix, Arizona, United States
- David Geffen School of Medicine at UCLA, Los Angeles, California, United States
- UCSF Benioff Children s Hospital, Oakland, California, United States
- Rady Children s Hospital, San Diego, California, United States
- University of Colorado School of Medicine, Aurora, Colorado, United States
- Connecticut Childrens Medical Cntr, Hartford, Connecticut, United States
- University of Chicago Medical Center, Chicago, Illinois, United States
- Minnesota Epilepsy Group, Saint Paul, Minnesota, United States
- Atlantic Health Systems, Morristown, New Jersey, United States
- Cinn Children Hosp Medical Center, Cincinnati, Ohio, United States
- ...and 10 more locations
Frequently Asked Questions
What is clinical trial NCT02962414?
NCT02962414 is a Phase 3 INTERVENTIONAL study titled "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." It is currently active, not recruiting and is sponsored by Novartis Pharmaceuticals. The trial targets enrollment of 206 participants.
What conditions does NCT02962414 study?
This trial investigates treatments for Tuberous Sclerosis Complex. The primary condition under study is Tuberous Sclerosis Complex.
What treatments are being tested in NCT02962414?
The interventions being studied include: everolimus (DRUG). everolimus, 2mg dispersible tablets
What does Phase 3 mean for NCT02962414?
Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.
What is the current status of NCT02962414?
This trial is currently "Active, Not Recruiting." It started on 2017-06-08. The estimated completion date is 2027-08-13.
Who is sponsoring NCT02962414?
NCT02962414 is sponsored by Novartis Pharmaceuticals. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT02962414?
The trial aims to enroll 206 participants. The trial status is active, not recruiting.
How is NCT02962414 designed?
This is a interventional study, uses na allocation, follows a single_group design, employs none masking.
What are the primary outcomes being measured in NCT02962414?
The primary outcome measures are: Occurances of adverse events and serious adverse events (Day 1 up to approximately 10 years). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT02962414 being conducted?
This trial is being conducted at 20 sites, including Phoenix, Arizona; Los Angeles, California; Oakland, California; San Diego, California and 16 more sites (United States, Australia, Belgium).
Where can I find official information about NCT02962414?
The official record for NCT02962414 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT02962414. This government database provides the most up-to-date and detailed information about the trial.
What is NCT02962414 testing in simple terms?
This study checks if everolimus is safe for long-term use in patients with TSC and seizures who have benefited from it. It's for patients who are currently in the EXIST-3 study and are getting better with everolimus.
Why is this trial significant?
This trial is crucial as it fills the gap in understanding the long-term safety of everolimus for TSC patients. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.
What are the potential risks of participating in NCT02962414?
Possible side effects include mouth sores, nausea, and changes in blood tests. Always report any new symptoms to your doctor. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT02962414?
Ask your doctor if you are currently in the EXIST-3 study and getting better with everolimus. You will take everolimus tablets and visit the doctor every 12 weeks for check-ups. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT02962414 signal from an investment perspective?
The large market size and Novartis' strong position make this a promising investment opportunity with a high approval probability. This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.
What happens if the treatment in this trial doesn't work?
Participants take everolimus tablets and visit the doctor every 12 weeks for check-ups. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.