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The effect of simvastatin on the cognitive deficits of children with neurofibromatosis I (NF1): a randomised, double-blind placebo-controlled study
ISRCTN ISRCTN14965707
DOI 10.1186/ISRCTN14965707
ClinicalTrials.gov identifier
EudraCT number
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.
Lay summary
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
  Email 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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