Defining Endpoints in Becker Muscular Dystrophy

Running, not enrolling

Conditions studied: Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker

In brief

This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

Key facts

Study ID

NCT05257473

Run by

Virginia Commonwealth University

People needed

80

Starts

2022-04-13

Expected to finish

2026-08-01

Last updated by the study team

2026-06-10

Who can join

Age: 6 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• For ages 6-12
• Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
• Genetic confirmation of an in-frame dystrophin mutation
• Ambulatory
• Willing and able to give informed consent and follow all procedures and requirements
• For ages 13 and older
• Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD)
• Genetic confirmation of a dystrophin mutation
• Willing and able to give informed consent and follow all procedures and requirements
• For participants in the MRI substudy:
• Ambulatory, defined as able to walk 10 meters without assistive devices (orthotics allowed)

You may not qualify if…

• For ages 6-12
• Out of frame dystrophin mutation
• Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
• Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
• >16 hours of ventilatory support
• Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
• Under the age of 6 at time of enrollment
• For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
• For ages 13 and older
• Loss of ambulation prior to age 16
• Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population
• Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics)
• >16 hours of ventilatory support
• Subjects aged 13-16 only: time to rise >10 seconds
• For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)

Where it is running

• University of California, Irvine — Orange, California, United States
• University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• University of Auckland — Auckland, New Zealand
• John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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