Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex (RAPIT)
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|ClinicalTrials.gov Identifier: NCT01730209|
Recruitment Status : Unknown
Verified May 2015 by M.C.Y. de Wit, MD PhD, Erasmus Medical Center.
Recruitment status was: Recruiting
First Posted : November 21, 2012
Last Update Posted : May 5, 2015
|Condition or disease||Intervention/treatment||Phase|
|Tuberous Sclerosis Complex TSC Related Cognitive Disability TSC Related Autism TSC Related Learning Problems||Drug: Everolimus Drug: Placebo||Phase 2 Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex|
|Study Start Date :||November 2012|
|Estimated Primary Completion Date :||November 2015|
|Estimated Study Completion Date :||November 2016|
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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|