Paving the way: new study aims to sharpen tools for LGMD R1 trials
NCT ID NCT05618080
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to validate these tools so future treatment trials can be more effective.
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 help design better clinical trials for LGMD R1 treatments by providing validated ways to measure disease progression.
- What could go wrong
- This is an observational study, not testing a treatment. It may not directly lead to a therapy, and results depend on participant adherence and MRI consistency.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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The Community Health Clinic, Inc.
Shipshewana, Indiana, 46565, United States
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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 Florida
Gainesville, Florida, 32610, United States
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University of Iowa Hospitals and Clinics
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, Department of Neurology
Minneapolis, Minnesota, 55455, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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