TRON

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

Tuberous sclerosis is a genetic disease that causes the growth of benign tumours, known as tubers, in many different places in the body including the skin, kidneys and brain. In the brain, these tubors are associated with epilepsy and problems in learning and information processing (also known as neurocognition).

Several studies in people with tuberous sclerosis have found that a medicine called Everolimus can reduce the size of the tubers in the brain, but we do not yet know if there will also be an improvement in learning and executive function in the patient. The TRON trial is designed to find out if treatment with Everolimus can improve neurocognitive problems in people with tuberous sclerosis.

In the trial, participants will be randomly allocated to take either Everolimus or placebo for a total of six months. Their neurocognition will be measured before they start taking the drug and again when they have stopped taking the medication.