Measuring a urine muscle-injury marker in children with muscular dystrophy
Tests urinary titin after stair walking in children with Duchenne or Becker muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Urinary Titin Biomarker in DMD
Key facts
- Where
- Philadelphia, United StatesSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 10 years old?
Source: NCT07332013, who can take part
Where in United States
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Children's Hospital of Philadelphia
- Trial code
- NCT07332013
- Record updated
- 8 April 2026
- Original description
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).Read full original descriptionShow less
Non-invasive Evaluation of Urinary Titin as an IND-enabling Biomarker for Use in Duchenne Muscular Dystrophy (DMD) Clinical Trials
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.
Inclusion: 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; 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: 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; Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices; Female patients; Parental/guardian unable to provide informed consent.
DMD/BMD Subject Inclusion/Exclusion Criteria.