Simple urine test could revolutionize DMD drug trials
NCT ID NCT07332013
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after activities like walking down stairs, and track daily activity with wearable sensors. The goal is to develop a non-invasive biomarker that could make future clinical trials smaller, faster, and more informative.
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 could provide a simple urine test to track muscle damage in DMD, making future drug trials faster and more accurate.
- What could go wrong
- This is an early observational study with only 50 participants, so the biomarker may not prove reliable enough for widespread use.
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.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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About 50 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2026
- Expected to finish
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Dec 2029
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.
- Ages
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2 to 10 years
- Sex
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Male participants only
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
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.
DMD/BMD Subject Inclusion/Exclusion Criteria Inclusion Criteria: 1. Ambulatory at screening 2. Genetically confirmed diagnosis of DMD/BMD 3. Parental/guardian permission (informed consent) for children. Child assent will also be obtained from patients ages 7 years old and older and deemed by the investigator to be neurodevelopmentally appropriate 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion Criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent Healthy Control Subject Inclusion/Exclusion Criteria Inclusion criteria: 1. Healthy children without DMD, BMD, or other significant chronic medical disease 2. Ambulatory at Screening, defined as able to walk independently without assistive devices 3. Parental/guardian permission (informed consent). Child assent will also be obtained from patients aged 7 years and older and deemed by the investigator to be neurodevelopmentally appropriate. 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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