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| [ Print-friendly version ] |
| The effect of simvastatin on the cognitive deficits of children with neurofibromatosis I (NF1): a randomised, double-blind placebo-controlled study |
| ISRCTN | ISRCTN14965707 |
| ClinicalTrials.gov identifier | |
| Public title | The effect of simvastatin on the cognitive deficits of children with neurofibromatosis I (NF1): a randomised, double-blind placebo-controlled study |
| Scientific title | |
| Acronym | NF1 simvastatin trial |
| Serial number at source | NTR542 |
| Study hypothesis | Statin-treatment has been shown to normalise 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. |
| Ethics approval | Ethics approval received from the local medical ethics committee. |
| Study design | Randomised, double-blind placebo-controlled study |
| Countries of recruitment | Netherlands |
| Disease/condition/study domain | Neurofibromatosis type 1 (NF1) |
| Participants - inclusion criteria |
1. Children aged between 8 and 16 years
2. NF1 diagnosis according to the criteria of the National Institutes of Health 3. Visiting the multidisciplinary NF1-outpatient clinic at the Erasmus MC-Sophia Children's Hospital 4. Informed consent |
| Participants - exclusion criteria |
1. Pathology of the central nervous system (CNS) (hydrocephalus, epilepsy, radiotherapy, neurosurgery etc.)
2. Deafness and/or severely impaired vision 3. Use of anti-epileptics and/or neuroleptics Additional exclusion criteria (under METC review as of 2nd May 2006): 4. Insufficient cognitive abilities to obtain a reliable score on a verbal IQ test (WISC-RN) 5. Contra-indications for simvastatin-treatment 6. Planned hospitalisation within three months after planned date of inclusion |
| Anticipated start date | 20/01/2006 |
| Anticipated end date | 01/03/2007 |
| Status of trial | Completed |
| Patient information material | |
| Target number of participants | 60 |
| Interventions | Simvastatin (10 mg/day for month 1, 20 mg/day month 2, 20 mg/day month 3 for children 8-12 years old or 40 mg/day month 3 for children 12-16 years old) or placebo once a day. |
| Primary outcome measure(s) |
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 neurophysiological tests on adaptation of eye movements after 1 and 3 months (saccade-adaptation test, adaptation of eye-hand coordination) 3. Measurement of the size, number, localization and spectra of unidentified bright objects (UBOs), hyperintensities on T2 weighed magnetic resonance imaging (MRI) and 3D CSI 1H magnetic resonance spectroscopy (MRS) after 3 months |
| Secondary outcome measure(s) |
1. Score on the following neuropsychological tests after 1 and 3 months (after 1 month = under METC review as of 02 May 2006):
1.1. Judgement of line orientation test 1.2. Rey Complex Figure Test (copy) 1.3. Beery VMI Test 2. Score on the following neuropsychological tests after 3 months: 2.1. IQ-test: WISC-RN 2.2. Verbal Fluency Test 2.3. Trailmaking Test A&B 2.4. Wisconsin Card Sorting Test 2.5. Peabody Picture Vocabulary Test 2.6. Boston Naming Test 2.7. 15 Word-Test 2.8. Stroop Color Word Test 3. Identification of facial emotions (ANT). Outcome of the following questionnaires after 3 months: 3.1. Child Behavior Check List (CBCL parents) 3.2. Teacher Report form (TRF) 3.3. Child Behavior Check List (CBCL child) 3.4. Quality of Life Questionnaire CHQ-CF87 Dutch edition (child) (under METC review as of 02 May 2006) 3.5. Quality of Life Questionnaire CHQ-PF50 Dutch edition (parents) (under METC review as of 02 May 2006) 4. Performance on the following neurophysiological tests after 1 and 3 months: 4.1. Basic saccade performance 4.2. Smooth pursuit |
| Sources of funding | Sophia Children's Hospital Fund (The Netherlands) |
| Trial website | |
| Publications | Results in http://www.ncbi.nlm.nih.gov/pubmed/18632543 |
| Contact name | Dr L.C. Krab |
| Address |
Erasmus Medical Center
Department of Neurosciences (Ee 12.28) P.O. Box 1738 |
| City/town | Rotterdam |
| Zip/Postcode | 3000 DR |
| Country | Netherlands |
| Tel | +31 (0)10 4087337 |
| Fax | +31 (0)10 4089459 |
| l.krab@erasmusmc.nl | |
| Sponsor | Erasmus Medical Center (The Netherlands) |
| Address |
Department of Neurosciences
P.O. Box 1738 |
| City/town | Rotterdam |
| Zip/Postcode | 3000 DR |
| Country | Netherlands |
| Sponsor website: | http://www.erasmusmc.nl/content/englishindex.htm |
| Date applied | 14/02/2006 |
| Last edited | 25/07/2008 |
| Date ISRCTN assigned | 14/02/2006 |
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