NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate

Study Overview
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Brief Summary
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Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. We, the researchers at Hospices Civils de Lyon, designed a randomized, double blind, placebo controlled, crossover trial with a total follow-up duration of 9 weeks to evaluate the effect of methylphenidate (MPH) on the improvement on the simplified parents Conners' Rating Scale. In a parallel exploratory study we will compare the nature of attention deficit disorders in NF1 children to 30 ADHD NF1-free controls. Children aged 7 to 12 years are eligible when their intelligence quotient (IQ) is between 80 and 120. Fifty subjects (25 for each period) were required for testing the primary study hypothesis.

Official Title
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Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo

Conditions
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Neurofibromatosis Type 1

Intervention / Treatment
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* Drug: methylphenidate


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 7-12 years IQ between 80-120. Gender: male or female Children with neurofibromatosis type 1 (according to the National Institutes of Health [NIH] 1988). Patients with school difficulties pointed out by parents or teachers Patients with attention difficulties as defined by anamnesis Exclusion Criteria: IQ > 120 or IQ < 80 Child depression Unwillingness to participate Patients with cerebral complication of neurofibromatosis type 1 (chiasma glioma, moya-moya) as detected by cerebral magnetic resonance imaging (MRI). Participation in another study

Ages Eligible for Study
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Minimum Age: 7 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: methylphenidate|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days. |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the efficacy of methylphenidate compared to placebo on specific neuropsychologic, depression and anxiety scales |  |  | 
| To compare the nature of attention deficit disorders in NF1 children with those with primary ADHD, depression, and anxiety using the State Trait Anxiety Inventory for Children (STAI-C) and the Children's Depression Inventory (CDI). |  |  | 
| Time of measurements are realised on day 0, day 28, day 63 + 2 days. |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neurofibromatosis type 1, Attention deficit with hyperactivity disorder school difficulties, methylphenidate