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

Phase 3

Active, not recruiting

Brief 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

Detailed 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.

Recruitment & Eligibility

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.

Arm && Interventions

Group	Intervention	Description
everolimus	everolimus	everolimus, 2mg dispersible tablets

Primary Outcome Measures

Name	Time	Method
Occurances of adverse events and serious adverse events	Day 1 up to approximately 10 years	The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)

Secondary Outcome Measures

Name	Time	Method
Percentage of patients with clinical benefit	Day 1 up to approximately 10 years, assessed every 12 weeks,	At every quarterly visit (every 12 weeks ± 2 weeks), the Investigator is required to confirm that the patients continue to have clinical benefit as per Investigator's assessment.

Locations (15): 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
Oregon Health Sciences University
🇺🇸
Portland, Oregon, United States
Childrens Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Texas Scottish Rite Hos for Child
🇺🇸
Dallas, Texas, United States
Texas Childrens Hospital
🇺🇸
Houston, Texas, United States
Novartis Investigative Site
🇬🇧
York, United Kingdom