NCT07332013Recruiting
Urinary Titin Biomarker in DMD
Non-invasive Evaluation of Urinary Titin as an IND-enabling Biomarker for Use in Duchenne Muscular Dystrophy (DMD) Clinical Trials
Source: ClinicalTrials.gov · Last updated 8 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Interventional
- Enrollment
- 50 (estimated)
- First submitted
- 6 Nov 2025
- Start date
- 4 Mar 2026
- Primary completion date
- 31 Dec 2029
- Lead sponsor
- Children's Hospital of Philadelphia
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT07332013
What you commit to
- Study type: Interventional
- Planned participants: 50
- From 4 Mar 2026 to the primary completion date, 31 Dec 2029
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 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
Who the study is looking for
- 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
Criteria the registry did not label
- DMD/BMD Subject Inclusion/Exclusion Criteria
Age in the registry: 2 years to 10 years · Sex: Male · Accepts healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.