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

Condition(s) targeted: Neurofibromatosis Type 1

Intervention: methylphenidate (Drug)

Phase: Phase 4

Status: Recruiting

Sponsored by: Hospices Civils de Lyon

Official(s) and/or principal investigator(s):
Laurence LION-FRANCOIS, MD, Principal Investigator, Affiliation: Hospices Civils de Lyon

Overall contact:
Laurence LION-FRANCOIS, MD, Phone: 33 4 72 38 55 54, Email: laurence.francois@chu-lyon.fr

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

Study design: Randomized, Double-Blind, Placebo Control, Crossover Assignment

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

Secondary outcome:

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.

Minimum age: 7 Years. Maximum age: 12 Years. Gender(s): Both.

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

Laurence LION-FRANCOIS, MD, Phone: 33 4 72 38 55 54, Email: laurence.francois@chu-lyon.fr

Laurence LION-FRANCOIS, Lyon 69005, France; Recruiting
Laurence LION-FRANCOIS, MD, Phone: 33 4 72 38 55 54, Email: laurence.francois@chu-lyon.fr

Starting date: January 2004
Last updated: October 3, 2007