Can we measure the progression of childhood myotonic dystrophy well enough to test new therapies?
NCT ID NCT06747884
First seen Aug 06, 2026 · Last updated Aug 12, 2026 · Updated 3 times
Summary
This study follows children with congenital or childhood myotonic dystrophy over time to see how their symptoms change. Researchers will test a range of assessments, from walking speed and hand strength to language and quality of life, to find the best ways to measure the disease. The goal is to prepare for future clinical trials by identifying reliable tests and biological samples that can show whether a new treatment is working.
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
- This study could help design better clinical trials for pediatric myotonic dystrophy, potentially speeding up the development of treatments that improve mobility, speech, and quality of life in affected children.
- What could go wrong
- As a natural history study, it does not test any treatment, so there is no direct benefit to participants. The findings may not fully capture the variability of the disease, and improvements in trial design do not guarantee that future treatments will be effective.
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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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Arkansas Children's Hospital
NOT_YET_RECRUITINGLittle Rock, Arkansas, 72202, United States
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Hospital das Clínicas, University of São Paulo School of Medicine (HCFMUSP)
NOT_YET_RECRUITINGCerqueira César, São Paulo, 05403-010, Brazil
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University of Kansas Medical Center
NOT_YET_RECRUITINGKansas City, Kansas, 66160, United States
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Virginia Commonwealth University
RECRUITINGRichmond, Virginia, 23298, United States
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