Clinical trial NCT00169611: NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate
Official title: Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Neurofibromatosis Type 1
Keywords: Neurofibromatosis type 1
Attention deficit with hyperactivity disorder school difficulties
methylphenidate
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2005-09-15
Enrollment: 80
Sex: ALL
Minimum age (years): 7
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days.
Secondary outcomes:
measure: To evaluate the efficacy of methylphenidate compared to placebo on specific neuropsychologic, depression and anxiety scales
measure: 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).
measure: Time of measurements are realised on day 0, day 28, day 63 + 2 days.
Other outcomes:
Locations:
city: Lyon
country: France
facility: Laurence LION-FRANCOIS
Study officials:
affiliation: Service de Neuropédiatrie - Hôpital Femme Mère Enfant - Hospices Civils de Lyon - 59 bd Pinel - 69677 BRON - France
name: Laurence LION-FRANCOIS, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Service de Neuropédiatrie - Hôpital Armand Trousseau - PARIS - France
name: Isabelle KEMLIN
role: PRINCIPAL_INVESTIGATOR
References:
citation: Lion-Francois L, Gueyffier F, Mercier C, Gerard D, Herbillon V, Kemlin I, Rodriguez D, Ginhoux T, Peyric E, Coutinho V, Breant V, des Portes V, Pinson S, Combemale P, Kassai B; Reseau NF1 Rhone Alpes Auvergne-France. The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial. Orphanet J Rare Dis. 2014 Sep 10;9:142. doi: 10.1186/s13023-014-0142-4.