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

• Status: Active, not recruiting
• Trial end date: 2027-08-04
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

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.