Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex
Randomized double-blind placebo controlled intervention study in children with TSC between
age 4 and 15 years with an intelligence quotient (IQ) estimated <80 and/or special schooling
and/or autism spectrum disorder and/or learning disability requiring remedial teaching.
Patients are randomised to receive everolimus or placebo during a period of 12 months.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Cognitive ability measured by IQ
Assessed by Wechsler scales: Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III NL) and Wechsler Intelligence Scale for Children (WISC-III-NL)
M.C.Y. de Wit, MD. PhD.
Erasmus Medical Center Rotterdam
Netherlands: Medical Ethics Review Committee (METC)