Neurofibromatosis Type 1COGTRAINNCT02944032
Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
A study testing CogmedRM and Mobymax in people with Neurofibromatosis Type 1, run by Kristina Hardy at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 to 16 years
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- CogmedRM and Mobymax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 103 participants
- Where
- 1 siteUnited States
- Study dates
- May 2016to 27 Sep 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 8-16 years old at time of screening
- NF1 Diagnosis based on National Institute of Health (NIH) criteria
- Has an identified caregiver who is willing and able to oversee the training practice during the intervention period
- Has access to a telephone and phone number where they can be reached
- Both patient and caregiver have reading, speaking, and listening comprehension of English
- 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.
- >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.
Who cannot join
- 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
- Current treatment for intracranial lesions, progressive tumors as per MRI evaluation or treatment with chemotherapy within the past 6 months
- A motor, visual, or auditory handicap that prevents computer use
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Health System
Washington D.C., District of Columbia, 20010
About this study
Official title: Multimodal Intervention Trial for Cognitive Deficits in Neurofibromatosis Type 1: Efficacy of Computerized Cognitive Training and Stimulant Medication
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.
Read the 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.
- Conditions
- Neurofibromatosis Type 1
- Being tested
- CogmedRM, Mobymax
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CogmedRMOther
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.
MobymaxOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Cogmed | Experimental | 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. Receives: Other: CogmedRM |
| MobyMax | Active comparator | 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. Receives: Other: Mobymax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in CogState One-back subtest
Computerized task of working memory
Measured: From baseline to end of training, up to 11 weeks
Secondary outcomes (10)
Change in Attention Deficit Hyperactive Disorder- Rating Scale
Parent-completed rating scale
Measured: From baseline to end of training, up to 11 weeks
Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Spatial Span Backward
Visual-spatial working memory task
Measured: From baseline to end of training, up to 11 weeks
Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Digit Span Backward
Auditory working memory task
Measured: From baseline to end of training, up to 11 weeks
CogState One-card Learning
Computerized attention and executive functioning tasks
Measured: From baseline to end of training, up to 11 weeks
Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Working Memory Index
Parent-completed questionnaire
Measured: From baseline to end of training, up to 11 weeks
Change in Test of Word Reading Efficiency-Second Edition (TOWRE-2) Scores
Child-completed reading task
Measured: From baseline to end of training, up to 11 weeks
Change in Test of Everyday Reading Comprehension (TERC) Scores
Child-completed reading task
Measured: From baseline to end of training, up to 11 weeks
Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Metacognition Index
Parent-completed questionnaire
Measured: From baseline to end of training, up to 11 weeks
CogState Groton Maze Learning Task
Computerized task of executive functioning
Measured: From baseline to end of training, up to 11 weeks
Change in Wechsler Intelligence Scale for Children-Fifth Edition-Integrated Letter-Number Sequencing
Auditory working memory task
Measured: From baseline to end of training, up to 11 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2016 (estimated)
- Study startMay 2016
- Primary completion27 Sep 2021
- Study completion27 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 103 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kristina Hardy
- Sponsor type
- University, hospital or other
- Collaborators
- Children's National Research Institute, Royal Children's Hospital, Children's Hospital Los Angeles, Sydney Children's Hospitals Network
- Responsible party
- Sponsor-investigator, Kristina Hardy, Children's National Research Institute
- Investigators
- Kristina Hardy, PhD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02944032
- Sponsor's protocol ID
- 00007343
- Acronym
- COGTRAIN
- Status last verified
- Mar 2022
- First submitted
- 25 May 2016
- Last updated
- 3 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multimodal Intervention Trial for Cognitive Deficits in Neurofibromatosis Type 1: Efficacy of Computerized Cognitive Training and Stimulant Medication
- ClinicalTrials.gov ID
- NCT02944032
- Acronym / sponsor ID
- COGTRAIN / 00007343
- Conditions
- Neurofibromatosis Type 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 103 participants (actual)
- Lead sponsor
- Kristina HardyUniversity, hospital or other
- Collaborators
- Children's National Research Institute, Royal Children's Hospital, Children's Hospital Los Angeles, Sydney Children's Hospitals Network
- Study start
- May 2016 (actual)
- Primary completion
- 27 Sep 2021 (actual)
- Study completion
- 27 Sep 2021 (actual)
- First posted
- 25 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cogmed | Experimental | Other: CogmedRMCogmed 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. |
| MobyMax | Active comparator | Other: MobymaxMobyMax'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. |
Who may qualify
- Ages
- 8 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 8-16 years old at time of screening
- NF1 Diagnosis based on National Institute of Health (NIH) criteria
- Has an identified caregiver who is willing and able to oversee the training practice during the intervention period
- Has access to a telephone and phone number where they can be reached
- Both patient and caregiver have reading, speaking, and listening comprehension of English
Key exclusion criteria (4 listed)
- 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
- Current treatment for intracranial lesions, progressive tumors as per MRI evaluation or treatment with chemotherapy within the past 6 months
- A motor, visual, or auditory handicap that prevents computer use
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CogmedRMCogmed 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. | Other | Cogmed |
| MobymaxMobyMax'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. | Other | MobyMax |
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
10 secondary outcomes
Change in Attention Deficit Hyperactive Disorder- Rating Scale
Time frame: From baseline to end of training, up to 11 weeks
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
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
CogState One-card Learning
Time frame: From baseline to end of training, up to 11 weeks
Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Working Memory Index
Time frame: From baseline to end of training, up to 11 weeks
Change in Test of Word Reading Efficiency-Second Edition (TOWRE-2) Scores
Time frame: From baseline to end of training, up to 11 weeks
Change in Test of Everyday Reading Comprehension (TERC) Scores
Time frame: From baseline to end of training, up to 11 weeks
Change in Behavior Rating Inventory of Executive Functioning (BRIEF) Metacognition Index
Time frame: From baseline to end of training, up to 11 weeks
CogState Groton Maze Learning Task
Time frame: From baseline to end of training, up to 11 weeks
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kristina Hardy, PhD principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CogmedRM and Mobymax in people with Neurofibromatosis Type 1, run by Kristina Hardy at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in CogState One-back subtest (time frame: From baseline to end of training, up to 11 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2022.