TRON (version 1.0)

• Research type

  Research Study

• Full title

  TRON: A randomised, double blind, placebo-controlled study of RAD001 (Everolimus) in the treatment of neurocognitive problems in tuberous sclerosis.

• IRAS ID

  87031

• Contact name

  Julian Sampson

• Sponsor organisation

  Cardiff University, Research and Commercial Division

• Eudract number

  2011-004854-25

• ISRCTN Number

  000000000

• Research summary

  This research study is investigating whether the study drug (Everolimus) is safe and effective for the treatment of neurocognitive difficulties in people who have tuberous sclerosis (TSC). Studies in people with TSC have shown that Everolimus is effective in treating other complications of the condition, including growths in the brain and is already used to prevent organ rejection in transplant patients and to treat kidney cancer. It is not known however whether Everolimus is effective for treating other brain-related problems in people with TSC, including learning and thinking problems and epilepsy. This study is designed to provide some initial answers to this question. Individuals from around the UK who are aged 16 to 60 years with TSC and an IQ of more than 60 will be invited to take part. If they are eligible, 48 individuals will be randomly allocated in a ratio of 2:1 to receive either Everolimus or Placebo for 6 months continuously. In total, patients will visit University Hospital Wales 8 times over a 36 week period in order to complete a series of tests. These include various blood and urine tests and physical and neurological exams. Three months after they have finished receiving either Everolimus or Placebo, each patient will undergo a further assessment and will then exit the study.

• REC name

  Wales REC 3

• REC reference

  11/WA/0308

• Date of REC Opinion

  9 Nov 2011

• REC opinion

  Further Information Favourable Opinion