Trial of Everolimus in the treatment of neurocognitive problems in TSC

The TRON study is now open to volunteers with tuberous sclerosis. TRON will assess the effects of Everolimus (also known as Votubia), a Rapamycin-like drug, on memory and thinking and will also look for effects on epilepsy.

Why is this trial being done?

Recent trials have shown that Everolimus is effective in shrinking kidney growths (angiomyolipomas) and brain tumours (SEGAs) in many people with tuberous sclerosis. This trial will explore whether the drug is also effective for some of the major common effects on tuberous sclerosis on the brain – thinking and epilepsy.

Who can take part?

People with tuberous sclerosis aged 16 to 60 years if they:

  • Have no seizures (fits) at present or do have seizures but have not changed their epilepsy drugs in the 6 months before starting the trial
  • AND
  • Are able to participate in the memory and thinking test

(We will assess this before start in the trial, but people with severe learning difficulties will usually not be able to take part).

If I take part, what will I have to do?

Everyone who takes part will come to the University Hospital in Cardiff 8 times over almost 40 weeks. However, some people who wish to take part may not be able to do so if we find certain health problems at the first visit. The TSA have provided funds to cover travel costs for this and, if required, overnight accommodation costs for volunteers and a family member or carer.

Participants will receive either Everolimus or Placebo (an identical looking “sugar pill” with no active medicine). There is a 2 out of 3 chance of being treated with Everolimus and a 1 out of 3 chance of being treated with placebo. Volunteers will have medical checks including blood tests. They will also complete tests of memory and thinking that are like quizzes and computer games.

This video explains a TRON participant's experience of the study:

For further information on the study or to discuss potential eligibility please contact:

Dr Anurag Saxena

Clinical Research Fellow,
Institute of Medical Genetics, Cardiff University
Heath Park, Cardiff, CF14 4XN

Professor Julian Sampson

Clinical Professor of Medical Genetics,
Institute of Medical Genetics, Cardiff University
Heath Park, Cardiff. CF14 4XN

Contact details...

Phone: +44 02920746412 / 02920687608 / Fax: +44 029 20746551
Webpage: Tron Trials