| - candidate number | 1864 |
| - NTR Number | NTR542 |
| - ISRCTN | ISRCTN14965707 |
| - Date ISRCTN created | 14-feb-2006 |
| - date ISRCTN requested | 13-jan-2006 |
| - Date Registered NTR | 28-nov-2005 |
| - Secondary IDs | N/A |
| - Public Title | The effect of simvastatin on the cognitive deficits of children with Neurofibromatosis I (NF1): a randomized, double-blind placebo-controlled study. |
| - Scientific Title | The effect of simvastatin on the cognitive deficits of children with Neurofibromatosis I (NF1): a randomized, double-blind placebo-controlled study. |
| - ACRONYM | NF1 simvastatin trial |
| - hypothesis | Statin-treatment has been shown to normalize the learning- and attention deficits in NF1 +/- mice by decreasing Ras activity. The fact that statins are effective in NF1 mice, combined with their very good safety profile, makes them an ideal candidate drug to treat cognitive impairments associated with NF1 in human patients. |
| - Healt Condition(s) or Problem(s) studied | Neurofibromatosis type 1 (NF1) |
| - 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 | 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. |
| - mec approval received | yes |
| - multicenter trial | no |
| - randomised | yes |
| - masking/blinding | Double |
| - control | Placebo |
| - group | Parallel |
| - Type | 2 or more arms, randomized |
| - Studytype | intervention |
| - planned startdate | 20-jan-2006 |
| - planned closingdate | 1-mrt-2007 |
| - Target number of participants | 60 |
| - 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. |
| - Primary outcome | 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 outcome | 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. |
| - Timepoints | N/A |
| - Trial web site | N/A |
| - status | stopped: trial finished |
| - CONTACT FOR PUBLIC QUERIES | Drs. L.C. Krab |
| - CONTACT for SCIENTIFIC QUERIES | Dr. Y. Elgersma |
| - Sponsor/Initiator | Erasmus Medical Center, Department of Neurosciences |
| - Funding (Source(s) of Monetary or Material Support) | Sophia Children’s Hospital Fund |
| - Publications | http://www.ncbi.nlm.nih.gov/pubmed?term=18632543 |
| - Brief summary | Recent research has shown that the cognitive phenotype of NF1 +/- mice can be reversed by the administration of statins. Because the majority of children with NF1 suffer from learning disabilities, statins could potentially make a large difference in the morbidity associated with NF1. In a double-blind, randomized placebo-controlled trial, 60 children with NF1 are treated with simvastatin or placebo for three months. The effect of simvastatin treatment will be evaluated using neuropsychologic, neurophysiologic and radiologic parameters. |
| - Main changes (audit trail) | |
| - RECORD | 28-nov-2005 - 23-jul-2012 |
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