Summary

for people ages 2-65 (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

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

Official 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.

Details

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.

Keywords

Tuberous Sclerosis Complex Everolimus TSC Refractory seizure Roll over study Long term safety PASS Sclerosis Seizures Tuberous Sclerosis Sirolimus

Eligibility

For people ages 2-65

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

  • UCSF Benioff Children s Hospital in progress, not accepting new patients
    Oakland California 94609 United States
  • David Geffen School of Medicine at UCLA in progress, not accepting new patients
    Los Angeles California 90005-1752 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Novartis Pharmaceuticals
ID
NCT02962414
Phase
Phase 3
Study Type
Interventional
Last Updated
July 6, 2018