New study aims to sharpen tools for tracking becker muscular dystrophy
NCT ID NCT05257473
First seen Jun 25, 2026 · Last updated Sep 04, 2026 · Updated 3 times
Summary
This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which tests are most useful for tracking BMD in future clinical trials.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this study could identify the most reliable ways to measure disease progression in Becker muscular dystrophy, helping design better future treatment trials.
- What could go wrong
- This is an observational study, not a treatment trial. It will not test any therapy, and the findings may not apply to all patients or lead directly to new treatments.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2022
- Expected to finish
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Sep 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Individuals age 6+ who are clinically affected with Becker Muscular Dystrophy
- Ages
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6 years and older
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: For ages 6-12 1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD) 2. Genetic confirmation of an in-frame dystrophin mutation 3. Ambulatory 4. Willing and able to give informed consent and follow all procedures and requirements For ages 13 and older 1. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with BMD) 2. Genetic confirmation of a dystrophin mutation 3. Willing and able to give informed consent and follow all procedures and requirements For participants in the MRI substudy: 1\. Ambulatory, defined as able to walk 10 meters without assistive devices (orthotics allowed) Exclusion Criteria: For ages 6-12 1. Out of frame dystrophin mutation 2. Use of chronic corticosteroids at baseline, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population 3. Non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics) 4. \>16 hours of ventilatory support 5. 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. 6. Under the age of 6 at time of enrollment 7. 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 1. Loss of ambulation prior to age 16 2. Use of chronic corticosteroids, defined as greater than 6 months of chronic use, will be limited to 20% of the overall population 3. Less than 30% of the overall population will be non-ambulatory, defined as the inability to walk 10 meters without assistive device (excluding orthotics) 4. \>16 hours of ventilatory support 5. Subjects aged 13-16 only: time to rise \>10 seconds 6. For MR Cohort: Have contraindications to MRI or MRS (e.g., non-MR compatible implanted medical devices or severe claustrophobia)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, NE1 4EP, United Kingdom
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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University of Auckland
Auckland, New Zealand
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University of California, Irvine
Orange, California, 92868, United States
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University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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