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

• ClinicalTrials.gov Identifier: NCT00169611
• Recruitment Status: Completed
• First Posted: September 15, 2005
• Last Update Posted: January 27, 2011
• Sponsor: Hospices Civils de Lyon
• Information provided by: Hospices Civils de Lyon

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Neurofibromatosis Type 1	Drug: methylphenidate	Phase 4

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Double
• Official Title: Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo
• Study Start Date: January 2004
• Actual Primary Completion Date: January 2011
• Actual Study Completion Date: January 2011

Arms and Interventions

Outcome Measures

Primary Outcome Measures :

1. 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 :

1. To evaluate the efficacy of methylphenidate compared to placebo on specific neuropsychologic, depression and anxiety scales
2. 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).
3. Time of measurements are realised on day 0, day 28, day 63 + 2 days.

Eligibility Criteria

• Ages Eligible for Study: 7 Years to 12 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

France

Laurence LION-FRANCOIS

Lyon, France, 69005

Sponsors and Collaborators

Hospices Civils de Lyon

Investigators

• Principal Investigator: Laurence LION-FRANCOIS, MD; Service de Neuropédiatrie - Hôpital Femme Mère Enfant - Hospices Civils de Lyon - 59 bd Pinel - 69677 BRON - France
• Principal Investigator: Isabelle KEMLIN; Service de Neuropédiatrie - Hôpital Armand Trousseau - PARIS - France

More Information

Additional Information:

Related Info 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Lion-François L, Gueyffier F, Mercier C, Gérard D, Herbillon V, Kemlin I, Rodriguez D, Ginhoux T, Peyric E, Coutinho V, Bréant V, des Portes V, Pinson S, Combemale P, Kassaï B; Réseau NF1 Rhône 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.

• Responsible Party: Dr Laurence LION-FRANCOIS, Service de Neuropédiatrie - Hôpital Femme Mère Enfant -
• ClinicalTrials.gov Identifier: NCT00169611
• Other Study ID Numbers: 2003.310
• First Posted: September 15, 2005
• Last Update Posted: January 27, 2011
• Last Verified: January 2011

Keywords provided by Hospices Civils de Lyon:

Neurofibromatosis type 1; Attention deficit with hyperactivity disorder school difficulties; methylphenidate

Additional relevant MeSH terms:

Neurofibromatoses; Neurofibromatosis 1; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neuromuscular Diseases; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Methylphenidate; Central Nervous System Stimulants; Physiological Effects of Drugs; Dopamine Uptake Inhibitors; Neurotransmitter Uptake Inhibitors; Membrane Transport Modulators; Molecular Mechanisms of Pharmacological Action; Dopamine Agents; Neurotransmitter Agents