NCT01730209.

Trial name or title	Efficacy of RAD001/everolimus in autism and neuropsychological deficits in children with TSC (RAPIT)
Methods	Randomised double‐blind placebo controlled intervention study.
Participants	Children with TSC between age 4 and 15 years with an IQ estimated < 80 and/or special schooling and/or autism spectrum disorder and/or learning disability requiring remedial teaching.
Interventions	Participants are randomised into either: 1. placebo tablet OR 2. everolimus tablet (titrated to trough level 5 ‐ 10 ng/ml).
Outcomes	Primary outcomes: cognitive ability measured by IQ and Wechsler Intelligence Scale for Children (WISC‐III‐NL). Secondary outcomes: autistic features as assessed by Autism Diagnostic Observation Schedule (ADOS); social and communicational skills as assessed by social responsiveness scale (SRS) and Dutch Children's Communication Checklist (CCC‐2‐NL) questionnaires; working memory and attention, information processing as assessed by Cambridge Neuropsychological Test Automated Battery (CANTAB); visual‐motor integration as assessed by BEERY Visual‐Motor Integration (BEERY VMI), grooved pegboard; child behavior as assessed by Child Behavior Checklist (CBCL) and Teacher's Report Form (TRF) questionnaires; executive functioning as assessed by Behavior Rating Inventory of Executive Functioning (BRIEF) questionnaire Dutch version; sleeping problems as assessed by Sleep Disturbance Scale for Children (SDSC) questionnaire; child health as assessed by Child Health Questionnaire Parent Form (CHQ‐PF50) questionnaire; sensory related difficulties as assessed by Short Sensory Profile (SSP) questionnaire; epilepsy (comparison of epilepsy frequency during month previous to study start and last month of trial participation.EEG abnormalities). Other outcomes: school level as assessed by the school CITO (centraal instituut voor toetsontwikkeling) scores or reading and arithmetic scores; pharmacokinetics as assessed by measuring trough levels of everolimus; safety (levels of and abnormalities in blood control value).
Starting date	November 2012.
Contact information	M.C.Y. de Wit, MD. PhD. Tel: +31 10 703 6956. Email: tubereuzesclerose@erasmusmc.nl.
Notes	Sponsored by Erasmus Medical Center.