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…
ID
Bron
Verkorte titel
Aandoening
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.
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
1. Performance on neuropsychological tests on visuospatial memory and attention after 1 and 3 months (Rey Complex Figure test (recall), Bourdon Vos Test); <br>
2. Performance on neurofysiological tests on adaptation of movements after 1 and 3 months (saccade-adaptation test, adaptation of eye-hand coordination);<br>
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.
Achtergrond van het onderzoek
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.
Doel van het onderzoek
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.
Onderzoeksopzet
N/A
Onderzoeksproduct en/of interventie
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.
Algemeen / deelnemers
P.O. Box 1738
L.C. Krab
Rotterdam 3000 DR
The Netherlands
+31 (0)10 4087337
l.krab@erasmusmc.nl
Wetenschappers
P.O. Box 1738
L.C. Krab
Rotterdam 3000 DR
The Netherlands
+31 (0)10 4087337
l.krab@erasmusmc.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
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 .
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
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.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL499 |
| NTR-old | NTR542 |
| Ander register | : N/A |
| ISRCTN | ISRCTN14965707 |