Clinical Trial: Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Multimodal Intervention Trial for Cognitive Deficits in Neurofibromatosis Type 1: Efficacy of Computerized Cognitive Training and Stimulant Medication

Brief Summary: Th main objective of the study is to assess the efficacy of a home-based, computerized cognitive training (CT) program, called CogmedRM, targeted to improve working memory in children with NF1 and working memory difficulties.This is a Phase II randomized parallel group controlled clinical trial comparing two interventions on cognitive outcomes. Participants will be stratified by stimulant medication use and randomized equally between the two interventions within stratum. Participants will be in the study for to 11 weeks.

Detailed Summary:

Cognitive deficits are the most important cause of long-term dysfunction in patients with Neurofibromatosis type 1 (NF1). Among the most frequently-occurring difficulties are problems with attention, working memory, and executive functioning. Remediation and interventions to improve those deficits have the potential to impact the quality of life and long-term prognosis in this population. Cognitive training (CT) programs have increasingly been used independently or in conjunction with pharmacotherapies in children with accidental or disease-related brain injury. CogmedRM is both the most well-researched and widely-used CT program for remediation of WM deficits. Results from numerous randomized, controlled trials conducted with a variety of pediatric and adult patient populations generally show that CogmedRM training is associated with robust gains in performance-based WM scores over the short term, with some variability in improvement across disease groups. A single arm pilot study of CogmedRM in a sample of children with NF1 conducted at Children's National Medical Center has shown that the approach is likely feasible and acceptable to families.

Because many children with NF1 are treated with stimulant medications, and there is biological evidence that both CT and Methylphenidate act on dopaminergic systems, the investigators are also interested in examining whether or not there is a synergistic effect between these widely available and safe interventions. Thus, the aim is to assess the efficacy of a home-based, computerized cognitive training (CT) program in a sample of 90 children aged 8-16 with NF1 and working memory difficulties. This study will be conducted over the span of four years. If the participant qualifies following baseline testing, he/she will be randomized to the intervention, CogmedRM, or the active control condition, MobyMax (an online reading program).
Sponsor: Children's Research Institute

Current Primary Outcome: Change in CogState One-back subtest [ Time Frame: From baseline to end of training, up to 11 weeks ]

Computerized task of working memory


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in Attention Deficit Hyperactive Disorder- Rating Scale [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Parent-completed rating scale
  • Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Spatial Span Backward [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Visual-spatial working memory task
  • Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Digit Span Backward [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Auditory working memory task
  • CogState One-card Learning [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Computerized attention and executive functioning tasks
  • Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Working Memory Index [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Parent-completed questionnaire
  • Change in Test of Word Reading Efficiency—Second Edition (TOWRE-2) Scores [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Child-completed reading task
  • Change in Test of Everyday Reading Comprehension (TERC) Scores [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Child-completed reading task
  • Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Metacognition Index [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Parent-completed questionnaire
  • CogState Groton Maze Learning Task [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Computerized task of executive functioning
  • Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Letter-Number Sequencing [ Time Frame: From baseline to end of training, up to 11 weeks ]
    Auditory working memory task


Original Secondary Outcome: Same as current

Information By: Children's Research Institute

Dates:
Date Received: May 25, 2016
Date Started: May 2016
Date Completion: September 2019
Last Updated: March 13, 2017
Last Verified: March 2017