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

Novartis

Status and phase

Active, not recruiting

Phase 3

Conditions

Tuberous Sclerosis Complex

Treatments

Drug: everolimus

Study type

Interventional

Funder types

Industry

Identifiers

NCT02962414

CRAD001M2X02B

Details and patient eligibility

About

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

Full description

This is a multi-center, open label study to evaluate the long term safety of everolimus in patients with TSC and refractory seizures currently being treated in the Novartis-sponsored study EXIST-3 study and are judged by their parent study investigator as benefiting from the current study treatment. The study is expected to remain open for approximately 10 years (or until 31-Dec-2022 in the UK) from First Patient First Visit (FPFV).

Patients will continue to be treated in this study until they are no longer benefiting from their everolimus treatment as judged by the Investigator or until one of the protocol defined discontinuation criteria is met or everolimus becomes commercially available according to local regulations.

Enrollment

206 patients

Sex

All

Ages

2 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and 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.