Trial of Everolimus (RAD001) in the treatment of neurocognitive problems in patients with tuberous sclerosis
Tuberous sclerosis (TSC) is a genetic disorder affecting ~1 in 10,000 people and is characterised by the development of tumours in many organs, including the skin and kidneys, and by brain involvement leading to seizures, neurocognitive deficits and behavioural and developmental disorders.
These brain-related problems occur in the majority of those with TSC, and have a significant impact on both patients and their families because of their everyday impact on education, employment, family and social life. The potential benefits of better treatment therefore include both a reduction in health care demands and wider benefits for patients and their carers.
TRON is a, two-arm, double- blind trial for the use of everolimus versus a placebo in the treatment of neurocognitive problems in patients with TSC. The study will recruit 48 male and female TS patients aged between 16 and 60 years. Each patient will be in the study for 6 months and undergo neurocognitive and medical assessments.
A phase II Randomised Controlled Trial (IMP). The IMP is a licensed medicine in this patient group but for a different target of effect. The current trial is a proof of principle study for memory and executive function outcomes.
Novartis Pharmaceuticals Ltd.