The effect of simvastatin on the cognitive deficits of children with Neurofibromatosis I (NF1): a randomized, double-blind placebo-controlled study.

• Register: Netherlands Trial Register
• Last refreshed on: 28 April 2013
• Main ID: NTR542
• Date of registration: 28/11/2005
• Primary sponsor: Erasmus Medical Center, Department of Neurosciences
• 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. - NF1 simvastatin trial
• Date of first enrolment: 20/1/2006
• Target sample size: 60
• Recruitment status: complete
• URL: http://www.trialregister.nl/trialreg/admin/rctview.asp?TC=542
• Study type: intervention
• Study design: Randomised: Yes; Masking: Double; Control: Placebo; Group: Parallel; Type: 2 or more arms, randomized

Countries of recruitment

The Netherlands

Contacts

• Name: L.C. Krab

• Name: Y. Elgersma

Key inclusion & exclusion 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: 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.

Age minimum:
Age maximum:
Gender:

Health Condition(s) or Problem(s) studied

Neurofibromatosis type 1 (NF1)

Intervention(s)

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(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 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(s)

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.

Secondary ID(s)

ISRCTN14965707

N/A

Source(s) of Monetary Support

Sophia Children?s Hospital Fund