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 3Active Not RecruitingNCT02962414

Novartis Pharmaceuticals206 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

206

Conditions

Tuberous Sclerosis Complex

Interventions

Eligibility

Sex: ALLMin age: 2 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (68)

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

Outcomes

Primary Outcomes

Occurances of adverse events and serious adverse events

The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)

Time frame: Day 1 up to approximately 10 years

Secondary Outcomes

Percentage of patients with clinical benefit

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.

Time frame: Day 1 up to approximately 10 years, assessed every 12 weeks,