Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
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.
Conditions:
🦠 Duchenne Muscular Dystrophy
🗓️ Study Start (Actual) 2 February 2022
🗓️ Primary Completion (Estimated) December 2024
✅ Study Completion (Estimated) December 2024
👥 Enrollment (Estimated) 90
🔬 Study Type OBSERVATIONAL
📊 Phase N/A
Locations:
📍 Richmond, Virginia, United States

📋 Eligibility Criteria

Description

    Inclusion Criteria:

    • * 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

    Exclusion Criteria:

    • * 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.
Ages Eligible for Study: 3 Years to N/A (CHILD, ADULT, OLDER_ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 4 March 2022
  • First Submitted that Met QC Criteria 4 March 2022
  • First Posted 15 March 2022

Study Record Updates

  • Last Update Submitted that Met QC Criteria 15 April 2024
  • Last Update Posted 16 April 2024
  • Last Verified April 2024