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

Hospices Civils de Lyon80 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Masking

DOUBLE

Enrollment

Conditions

Neurofibromatosis Type 1

Interventions

Eligibility

Sex: ALLMin age: 7 YearsMax age: 12 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days.

Secondary Outcomes

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.