Actively Recruiting
Urinary Titin Biomarker in DMD
Led by Children's Hospital of Philadelphia · Updated on 2026-04-08
50
Participants Needed
1
Research Sites
195 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Philadelphia
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful functional improvement. This has been a particular challenge in rare and slowly progressive diseases such as Duchenne Muscular Dystrophy (DMD). The investigators hypothesize that urinary N-terminal fragment of titin (NTFT) corresponding to activity level/intensity will define a high-precision, non-invasive biomarker of systemic muscle injury to enable serial measurements of efficacy and safety in the clinical investigation of gene therapy for DMD and other myopathies. This should provide a valuable exploratory, secondary and eventually primary outcome measure of therapeutic efficacy to minimize the enrollment size in informative early phase and pivotal clinical trials.
CONDITIONS
Official Title
Urinary Titin Biomarker in DMD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male ambulatory children aged 2 to 10 years
- Genetically confirmed diagnosis of Duchenne Muscular Dystrophy or Becker Muscular Dystrophy
- Parental or guardian permission with informed consent; child assent for ages 7 and older when appropriate
- Access to electricity and a freezer at home to use devices and store samples
- Healthy ambulatory children without DMD, BMD, or other significant chronic diseases may also participate as controls with similar consent and home access requirements
You will not qualify if you...
- Non-ambulatory participants unable to walk independently and requiring assistive devices
- Female patients
- Parental or guardian unable to provide informed consent
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
C
Calliope O'Donnell, MS
CONTACT
J
Julianne Larosa, MPH
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
BASIC_SCIENCE
Number of Arms
1
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