Trial of Everolimus in the treatment of neurocognitive problems in TSC - TRON study


TRON study now recruiting for participants from across the UK to attend centres in Cardiff, Glasgow and Belfast

Funding is available to cover patient expenses (travel and overnight stay, if necessary) to enable participants to visit the TRON study centres in Cardiff, Glasgow and Belfast. The TSA is also very grateful to the Hospital Saturday Fund for providing a grant to allow us to provide this support.

The TRON study is now open to volunteers from across the UK with tuberous sclerosis. With centres in Cardiff, Glasgow and Belfast, people will be supported to attend the centres closest to where they live. (So this means if you live in the North of England you can attend the Glasgow centre).

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 attend the centre closest to them (Cardiff, Glasgow or Belfast) 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. Funds are available to support 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
Email: tron@cardiff.ac.uk
Webpage: Tron Trials


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