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The effect of long-term Simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial.


- candidate number6837
- NTR NumberNTR2150
- ISRCTNISRCTN wordt niet meer aangevraagd.
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR30-dec-2009
- Secondary IDsNL27196.000.09 CCMO
- Public TitleThe effect of long-term Simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial.
- Scientific TitleThe effect of long-term Simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized doubleblind placebo controlled trial.
- ACRONYMNF1-SIMCODA
- hypothesisSimvastatin can have a positive effect on cognitive function and daily life functioning in children with Neurofibromatosis 1.
- Healt Condition(s) or Problem(s) studiedNeurofibromatosis type 1 (NF1)
- Inclusion criteria1. Age 8 to 16 years;
2. NF1 diagnosis with genetic confirmation;
3. Oral and written informed consent from parents and participants aged older than 12 years.
- Exclusion criteria1. Segmental NF1;
2. Pathology of the CNS (other than asymptomatic gliomas);
3. Deafness;
4. Severely impaired vision;
5. Use of ADHD-medication;
6. Use of anti-epileptic drugs;
7. Use of anti-psychotic medication;
8. Use of Simvastatin;
9. Insufficient comprehension or production of the Dutch language;
10. An IQ below 48.
- mec approval receivedyes
- multicenter trialyes
- randomisedyes
- masking/blindingDouble
- controlPlacebo
- groupParallel
- Type2 or more arms, randomized
- Studytypeintervention
- planned startdate 1-mrt-2010
- planned closingdate1-mrt-2012
- Target number of participants106
- InterventionsParticipants are treated for 12 months with Simvastatin (10 mg/d in month 1, 20 mg/d in month 2, and then 20 mg/d for participants 8-12 years old or 40 mg/d for participants 13-16 years old) or equivalent placebo once a day in the morning).
- Primary outcome1. Cognitive Function: WISC III-R;
2. Behavioral Problems on the Child Behavioral Checklist (CBCL, parents): internalizing behavioral problems, attention problems.
- Secondary outcome1. School performance: Teacher ratings form the Teacher Report Form;
2. Quality of Life: CHQPF50 Child Quality of Life Questionnaire;
3. Behavior (other): internalizing problems rated by children on the Youth Self Report;
4. Cognitive Function (other): Non-verbal Long term Memory by the Rey Complex Figure test delayed recall;
5. Attention (other): Stroop color word test;
6. Fine motor coordination: Grooved Pegboard test.
- TimepointsBaseline (T=0 months) and end of treatment (T=12 months).
- Trial web siteN/A
- statusstopped: trial finished
- CONTACT FOR PUBLIC QUERIESdrs. M.H.T. Vaart, van der
- CONTACT for SCIENTIFIC QUERIESProf. dr. Ype Eigersma
- Sponsor/Initiator Erasmus Medical Center, Sophia Children's Hospital
- Funding
(Source(s) of Monetary or Material Support)
ZON-MW, The Netherlands Organization for Health Research and Development
- PublicationsN/A
- Brief summaryChildren with Neurofibromatosis type 1 (NF1, prevalence 1:3000) commonly display cognitive deficits that have a large impact on behaviour, school performance and quality of life. Mouse model studies showed that the learning deficits are due to increased RAS signalling. This increased signalling, as well as the synaptic plasticity deficits and learning deficits of these mice can be rescued by statin-mediated inhibition of HMG-CoA reductase. Statins are the most commonly prescribed drugs worldwide, and have a very favourable safety profile. Hence, this provides us with a unique opportunity to assess the effect of a targeted treatment on cognitive performance and behaviour in patients. We have previously conducted a 12-week pilot study in children with NF1 (ISRCTN14965707). Although we did not find a significant improvement compared to placebo in the primary outcome measures, we did obtain preliminary data that statins improved some measures. We consider it certainly possible that there is indeed a significant therapeutic benefit, but that the overall treatment was too short to detect this in the pilot trial. In addition, the clinical outcome measures may have been unable to reflect a potential benefit in daily life functioning. Furthermore, we found indications that ADHD-medication may mask the beneficial effect of Simvastatin on attention, thus patients that are treated with ADHD-medication will be excluded from this trial. This trial will aim to clarify the effect of long-term Simvastatin treatment on cognition and daily life functioning in children with NF1.
- Main changes (audit trail)19-Apr-2012: Inclusion has stopped since 8-Mar-2012. The target number of participants has been decreased from 106 to 84. Follow-up will take until March 2013 - NM
26-Apr-2012: Recruitment was stopped at 84 participants and did not reach the predetermined 106 subjects. Decision to stop inclusion was due to slow inclusion and not based on any knowledge of outcome. Main reason for slow recruitment was an unexpected high use of stimulant medication. The decision to stop inclusion at 84 participants results in overall expected power decrease of 90% to 85%, which we accept - NM
- RECORD30-dec-2009 - 13-mrt-2013


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