Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex (RAPIT)
Recruitment status was: Recruiting
|Tuberous Sclerosis Complex TSC Related Cognitive Disability TSC Related Autism TSC Related Learning Problems||Drug: Everolimus Drug: Placebo||Phase 2 Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex|
- Cognitive ability measured by IQ [ Time Frame: 12 months ]Assessed by Wechsler scales: Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III NL) and Wechsler Intelligence Scale for Children (WISC-III-NL)
- Autistic features [ Time Frame: 12 Months ]Assessed by Autism Diagnostic Observation Schedule (ADOS)
- Social and communicational skills [ Time Frame: 12 Months ]Assessed by social responsiveness scale (SRS) and Dutch Children's Communication Checklist (CCC-2-NL) questionnaires
- Working memory and attention, information processing [ Time Frame: 6 and 12 Months ]Assessed by Cambridge Neuropsychological Test Automated Battery (CANTAB)
- Visual-motor integration [ Time Frame: 12 Months ]Assessed by BEERY Visual-Motor Integration (BEERY VMI), grooved pegboard
- Child behavior [ Time Frame: 12 Months ]Assessed by Child Behavior Checklist (CBCL) and Teacher's Report Form (TRF) questionnaires
- Executive functioning [ Time Frame: 12 Months ]Assessed by Behavior Rating Inventory of Executive Functioning (BRIEF) questionnaire Dutch version
- Sleeping problems [ Time Frame: 12 Months ]Assessed by Sleep Disturbance Scale for Children (SDSC) questionnaire
- Child health [ Time Frame: 12 Months ]Assessed by Child Health Questionnaire Parent Form (CHQ-PF50) questionnaire
- Sensory related difficulties [ Time Frame: 12 Months ]Assessed by Short Sensory Profile (SSP) questionnaire
- Epilepsy [ Time Frame: 12 Months ]
Comparison of epilepsy frequency during month previous to study start and last month of trial participation.
- School level [ Time Frame: 12 Months ]Assessed by the school CITO (centraal instituut voor toetsontwikkeling) scores or reading and arithmetic scores
- Pharmacokinetics [ Time Frame: 12 Months ]Assessed by measuring trough levels of everolimus
- Safety [ Time Frame: 12 Months ]Levels of and abnormalities in blood control values
|Study Start Date:||November 2012|
|Estimated Study Completion Date:||November 2016|
|Estimated Primary Completion Date:||November 2015 (Final data collection date for primary outcome measure)|
Everolimus once daily for 1 year, titration to trough levels of 5-10 ng/ml
Everolimus once daily titrated to trough levels of 5-10 ng/ml.
Placebo Comparator: Placebo
Placebo treatment for 1 year. Tablets will be identical to everolimus tablets.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01730209
|Contact: M.C.Y. de Wit, MD. PhD.||+31 10 703 email@example.com|
|Erasmus Medical Center||Recruiting|
|Contact: M.C.Y. de Wit, MD. PhD. +31 10 703 6956 firstname.lastname@example.org|
|Principal Investigator: M.C.Y. de Wit, MD. PhD.|
|Sub-Investigator: I.E. Overwater, MSc|
|Principal Investigator:||M.C.Y. de Wit, MD. PhD.||Erasmus Medical Center, Rotterdam|