Neurofibromatosis type 1 is the most common single gene disease causing learning disabilities in humans. Children with NF1 commonly have cognitive dysfunctions like learning and attention deficits as well as impaired motor coordination. Half of the children seen at the multidisciplinary NF1 outpatient clinic of the Sophia Children's Hospital attends special education.
Conditions
Interventions
Simvastatin (10 mg/d for month 1, 20 mg/d month 2, 20 mg/d month 3 for children 8-12 years old or 40 mg/d month 3 for children 12-16 years old) or placebo once a day.
Sponsors
Erasmus MC afd. Neurowetenschappen, Dr. Y. Elgersma
Eligibility
Inclusion criteria
Inclusion criteria: Children aged between 8 and 16 years, NF1 diagnosis according to the criteria of the National Institutes of Health, visiting the multidisciplinary NF1-outpatient clinic at the Erasmus MC – Sophia Children’s Hospital; informed consent .
Exclusion criteria
Exclusion criteria: Pathology of the CNS (hydrocephalus, epilepsy, radiotherapy, neurosurgery, etc.), deafness and/or severely impaired vision, use of anti-epilepics and/or neuroleptics. Additional exclusion-criteria (under METC review): a. Insufficient cognitive abilities to obtain a reliable score on a verbal IQ test (WISC-RN); b. Contra-indications for simvastatin-treatment; c. Planned hospitalization within three months after planned date of inclusion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Performance on neuropsychological tests on visuospatial memory and attention after 1 and 3 months (Rey Complex Figure test (recall), Bourdon Vos Test); 2. Performance on neurofysiological tests on adaptation of movements after 1 and 3 months (saccade-adaptation test, adaptation of eye-hand coordination); 3. Measurement of the size, number, localization and spectra of UBO’s (unidentified bright objects, hyperintensities on T2 weighed MRI), on T2 MRI and 3D CSI 1H-MRS after 3 months. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Score on the following neuropsychological tests after 1 and 3 months (after 1 month = under METC review): 1a. Judgement of line orientation test; 1b. Rey Complex Figure Test (copy); 1c. Beery VMI Test; 2. Score on the following neuropsychological tests after 3 months: 2a. IQ-test: WISC-RN; 2b. Verbal Fluency Test; 2c. Trailmaking Test A&B; 2d. Wisconsin Card Sorting Test; 2e. Peabody Picture Vocabulary Test; 2f. Boston Naming Test; 2g. 15 Word-Test; 2h. Stroop Color Word Test; 3. Identification of facial emotions (ANT) Outcome of the following questionnaires after 3 months: 3a. Child Behavior Check List (CBCL parents); 3b. Teacher Report form (TRF); 3c. Child Behavior Check List (CBCL child); 3d. Quality of Life Questionnaire CHQ-CF87 Dutch edition (child) (under METC review); 3e. Quality of Life Questionnaire CHQ-PF50 Dutch edition (parents) (under METC review); 4. Performance on the following neurophysiological tests after 1 and 3 months: 4a. Basic saccade performance; 4b. Smooth pursuit. | — |
Contacts
Public ContactL.C. Krab
Erasmus Medical Center, Department of Neurosciences (Ee 12.28), P.O. Box 1738
Outcome results
None listed