Long term safety of Everolimus in patients with TSC related seizures

• Research type

  Research Study

• Full 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 (EXIST-3)

• IRAS ID

  221513

• Contact name

  Finbar O'Callaghan

• Contact email

  f.o'callaghan@ucl.ac.uk

• Sponsor organisation

  Novartis Pharmaceuticals UK Limited,

• Eudract number

  2016-002977-37

• Duration of Study in the UK

  5 years, 9 months, 5 days

• Research summary

  TSC is a genetic disorder affecting around one in 6,000 births; a total of approximately 8,000 people in the UK are living
  with the condition. It causes noncancerous
  tumours to form in vital organs, and can affect many different parts of the
  body, most commonly the brain and the kidney. As many as 90% of patients with tuberous sclerosis have seizures , a
  significant proportion of whom are resistant to medical therapy
  The goal of this study is to evaluate the long-term safety and to allow continued supply of everolimus to patients who are currently receiving everolimus treatment in the Novartis-sponsored study, EXIST-3, that has reached its study objectives. These patients are judged by the parent study Investigator as benefiting from continued treatment and should be unable to access everolimus outside of a clinical trial.

  Patients who are eligible to transition on to the roll over study will continue to receive everolimus and will be required to attend clinic every 12 weeks for monitoring and dispensing of study drug. About 200 patients from 66 centres around the world will participate in this new study.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  17/LO/0237

• Date of REC Opinion

  27 Feb 2017

• REC opinion

  Unfavourable Opinion