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

• Register: ClinicalTrials.gov
• Last refreshed on: 1 April 2024
• Main ID: NCT02962414
• Date of registration: 09/11/2016
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: 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
• Scientific title: 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.
• Date of first enrolment: June 8, 2017
• Target sample size: 206
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02962414
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Colombia, France, Hungary, Italy, Japan, Korea, Republic of, Mexico, Poland, Russian Federation, Spain, Taiwan, Thailand, Turkey, United Kingdom, United States

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Tuberous Sclerosis Complex

Intervention(s)

Drug: everolimus

Primary Outcome(s)

Occurances of adverse events and serious adverse events [Time Frame: Day 1 up to approximately 10 years]

Secondary Outcome(s)

Percentage of patients with clinical benefit [Time Frame: Day 1 up to approximately 10 years, assessed every 12 weeks,]

Secondary ID(s)

CRAD001M2X02B

Source(s) of Monetary Support

Please refer to primary and secondary sponsors