An observational study in Duchenne Muscular Dystrophy, sponsored by Virginia Commonwealth University. Completed at 1 site in United States. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2025-08-14.
Sponsored by Virginia Commonwealth University · Observational
Duchenne Muscular Dystrophy is a genetic disease that causes progressive muscle weakness. There is now substantial evidence that boys with this disease do not demonstrate age-related gains in their cognitive skills.
The goals of this study are (i) to use a technology-enabled neurobehavioral assessment called National Institutes of Health Toolbox Cognition Battery (NIHTB-CB) to assess brain development over time; (ii) engage with key-stakeholders to understand how neurodevelopmental problems like attention-deficit hyperactivity, autism spectrum affects individuals (and/or) families, so that we can understand meaningful effects of a potential treatment at an individual level, and (iii) to investigate using brain magnetic resonance imaging (MRI) changes in brain connectivity.
Participants will be asked to do tasks such as solve puzzles, pay attention, and remember things through the NIHTB-CB administered using an IPad as well as complete questionnaires. The NIHTB-CB will be done four times (first visit, within 1 month after first visit, 12 months after first visit, and 18 months after first visit). NIHTB-CB takes about 20 to 35 minutes to complete.
Participants will also undergo brief assessment of the upper limbs; these tests will be administered by trained physical therapists and will take about 5 minutes.
Focus group: A focus group consisting of families and individuals affected by DMD will be invited to share their journey of cognitive and developmental needs. The focus group will be conducted online via the study team and is planned to last between 1 to 1.5 hours. This is optional for families.
A non-sedated brain MRI will be performed twice; scans will be separated by a one-year interval. (A child has to be age 8 or older to participate in a brain scan.) Brain imaging is optional for families.
473 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
This study's enrollment of 50 is below the median of 60 across 130 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Virginia Commonwealth University is the lead sponsor of 641 studies on the registry; 82 are open to participants now.
Of its 88 completed or terminated interventional studies of FDA-regulated products, 62 (70%) have results posted.
Counted across the registry records on this site, refreshed daily.
Eligible subjects will be recruited nationally.
Exclusion Criteria:
Boys ages 3 and above will be enrolled.
Other: No intervention
Healthy boys as a control group for brain imaging.
There is no intervention
Change in NIHTB-CB Total Cognition Score over time
Time frame: baseline and 18 months
Change in brain connectivity over time
Change in brain connectivity as measured by brain MRI
Time frame: baseline and 12 months
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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Virginia Commonwealth University