Brief Title
Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Official Title
Multimodal Intervention Trial for Cognitive Deficits in Neurofibromatosis Type 1: Efficacy of Computerized Cognitive Training and Stimulant Medication
Brief 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.
Study Type
Interventional
Primary Outcome
Change in CogState One-back subtest
Secondary Outcome
Change in Attention Deficit Hyperactive Disorder- Rating Scale
Condition
Neurofibromatosis Type 1
Intervention
CogmedRM
Study Arms / Comparison Groups
Cogmed
Description: 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. 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
130
Start Date
May 2016
Completion Date
September 2021
Primary Completion Date
September 2021
Eligibility Criteria
Inclusion Criteria:
1. 8-16 years old at time of screening
2. NF1 Diagnosis based on National Institute of Health (NIH) criteria
3. Has an identified caregiver who is willing and able to oversee the training practice
during the intervention period
4. Has access to a telephone and phone number where they can be reached
5. Both patient and caregiver have reading, speaking, and listening comprehension of
English
6. Treated with a stable dose of stimulant medication for at least the last 30 days and
not planning to change the dose during study participation or receiving no stimulant
medications for at least the last 30 days and not planning to initiate a trial of
stimulant medications for the duration of the study.
7. >1 Standard Deviation (SD) below the mean on the WISC-V-Integrated Spatial Span
Backwards task or a Spatial Span Backwards score >1 SD below the participant's
estimated IQ.
Exclusion Criteria:
1. Full scale IQ<70, as estimated by WASI-II (Block Design, Vocabulary, Matrix Reasoning,
Similarities).
Note: In cases where there is a statistically significant difference between verbal IQ
and performance IQ (.05 level as determined by the WASI-II manual), participants will
be eligible if at least one of these quotients is 70 or above
2. Current treatment for intracranial lesions, progressive tumors as per MRI evaluation
or treatment with chemotherapy within the past 6 months
3. A motor, visual, or auditory handicap that prevents computer use
Gender
All
Ages
8 Years - 16 Years
Accepts Healthy Volunteers
No
Contacts
Kristina Hardy, PhD, 202-476-2514, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02944032
Organization ID
00007343
Responsible Party
Sponsor-Investigator
Study Sponsor
Kristina Hardy
Collaborators
Children's National Health System
Study Sponsor
Kristina Hardy, PhD, Principal Investigator, Children's National Health System
Verification Date
March 2021