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\nin patients with tuberous sclerosis complex (TSC) and\nrefractory seizures who are judged by the Investigator to\nbenefit from continued treatment with everolimus after\ncompletion of Study CRAD001M2304 (EXIST-3)

• IRAS ID

  226490

• 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, 7 months, 31 days

• Research summary

  Patients who will be taking part in this study would be patients with Tuberous Sclerosis Complex (TSC) who have participated in the parent EXIST -3 study, whose study doctor has confirmed that they are benefitting from the study drug, Everolimus.\nTSC 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.\n\nThe goal of this rollover study is to evaluate the long-term safety and to allow continued supply of everolimus to patients who\nare currently receiving everolimus treatment in the Novartis-sponsored study, EXIST-3, which has reached its study\nobjectives.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  17/LO/0693

• Date of REC Opinion

  25 Aug 2017

• REC opinion

  Further Information Favourable Opinion