The use of everolimus in the treatment of neurocognitive problems in tuberous sclerosis (TRON): study protocol for a randomised controlled trial.

ABSTRACT:

Background

Tuberous sclerosis complex (TSC) is a genetic disorder affecting about 1 in 6000 people and is characterised by the development of tumours in many organs, including the skin and kidneys, and by a range of neurological and neuropsychiatric manifestations. TSC-associated neuropsychiatric disorders (TAND) occur in the majority of those with TSC, and they have a significant impact on patients and their families, given the everyday impact of TAND on education, employment, family and social life. The potential benefits of better treatment for TAND therefore include reduction in health care demands and wider benefits for patients and their carers.

Methods/design

We have planned a single-centre, two-arm, individually randomised, phase II, double-blind, placebo-controlled

SUBMITTER: Randell E 

PROVIDER: S-EPMC4981993 | biostudies-literature | 2016 Aug

REPOSITORIES: biostudies-literature