Urinary Titin Biomarker in DMD
Recruiting now · Not applicable
Conditions studied: Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
In brief
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.
Key facts
- Study ID
- NCT07332013
- Run by
- Children's Hospital of Philadelphia
- People needed
- 50
- Starts
- 2026-03-04
- Expected to finish
- 2029-12-01
- Last updated by the study team
- 2026-04-08
Who can join
Age: 2 and older, up to 10. Sex: male. Healthy volunteers: accepted.
You may qualify if…
- Ambulatory at screening
- Genetically confirmed diagnosis of DMD/BMD
- 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
- Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples
You may not qualify if…
- 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:
- Healthy children without DMD, BMD, or other significant chronic medical disease
- Ambulatory at Screening, defined as able to walk independently without assistive devices
- 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.
- 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
Where it is running
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.