Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy

Completed

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT05280730

Run by

Virginia Commonwealth University

People needed

50

Starts

2022-02-02

Expected to finish

2024-09-30

Last updated by the study team

2025-08-14

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Boys with confirmed genetic mutation in the dystrophin gene
• Boys with clinical features of DMD and in whom muscle biopsy showed absence of dystrophin
• Boys with clinical features of DMD and in whom there is a family history of DMD
• Symptomatic carrier girls with DMD
• Ages 3 and above at time of study screening

You may not qualify if…

• Care-giver unable to give consent
• Any handicap that does not allow the ability to use an IPAD
• For MRI, braces or any metal implants.

Where it is running

• Children's Hospital of Richmond — Richmond, Virginia, United States

Full record on ClinicalTrials.gov

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