Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Condition: Neurofibromatosis Type 1
MeSH terms: Neurofibromatoses; Neurofibromatosis 1; Neurofibroma
Study type: Interventional

Summary: 
      The 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 description: 
      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 (WM), and executive functioning (EF). 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). The participant will have 5-9 weeks to complete the program and
      will have follow-up testing 2 weeks after finishing the program. If CT, either singly or in
      combination with stimulant medication, can be shown to be efficacious in a sample of NF1
      pediatric patients at high risk for neurocognitive deficits, this intervention plan could be
      rapidly translated to clinical practice.
    

Intervention name(s): CogmedRM; Mobymax
Intervention type(s): Other; Other
Intervention description(s): Cogmed RM is a computer program that consists of twelve visually-engaging and interesting exercises that target skills involving visuo-spatial and verbal Working Memory. Difficulty of the tasks is automatically adjusted on a trial-by-trial basis throughout each training session to match a child's current working memory span, such that as the child becomes more proficient, the exercises become more difficult. During the intervention, children complete 25 training sessions. Children are asked to complete between 3 and 5 sessions per week, so the total treatment time to complete 25 sessions may range from 5 to 9 weeks. For children completing CogmedRM, sessions typically last between 25 and 45 minutes, depending on the child's working memory span.; MobyMax's "Reading Stories" program is a program that focuses on reading comprehension skills. Participants will start their training with stories that are matched to their reading "grade" level. Each grade contains 30 lessons, with 3 stories in each lesson. Participants are given questions to answer at the conclusion of each story, and children advance or remain at that level depending on the progression of their reading skill. Participants randomized to this program will be asked to train 30-45 minutes per session for 25 training sessions over a 5 to 9 week period.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Treatment