NCT00169611

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Jan 2004

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2004

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

September 9, 2005

Completed
6 days until next milestone

First Posted

Study publicly available on registry

September 15, 2005

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2011

Completed
Last Updated

January 27, 2011

Status Verified

January 1, 2011

Enrollment Period

7 years

First QC Date

September 9, 2005

Last Update Submit

January 26, 2011

Conditions

Neurofibromatosis Type 1

Keywords

Neurofibromatosis type 1Attention deficit with hyperactivity disorder school difficultiesmethylphenidate

Outcome Measures

Primary Outcomes (1)

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

Secondary Outcomes (3)

  • 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.

Interventions

methylphenidateDRUG

Eligibility Criteria

Age7 Years - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Laurence LION-FRANCOIS

Lyon, 69005, France

Location

Related Publications (1)

  • 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.

    PMID: 25205361DERIVED

Related Links

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Methylphenidate

Condition Hierarchy (Ancestors)

NeurofibromatosesNeurofibromaNerve Sheath NeoplasmsNeoplasms, Nerve TissueNeoplasms by Histologic TypeNeoplasmsNeoplastic Syndromes, HereditaryNeurocutaneous SyndromesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesPeripheral Nervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

PhenylacetatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsPiperidinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
DOUBLE
Intervention Model
CROSSOVER
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 9, 2005

First Posted

September 15, 2005

Study Start

January 1, 2004

Primary Completion

January 1, 2011

Study Completion

January 1, 2011

Last Updated

January 27, 2011

Record last verified: 2011-01

Locations

FR(1)