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Urinary Titin Biomarker in DMD

Children's Hospital of Philadelphia · with National Institute of Neurological Disorders and Stroke (NINDS) · NCT07332013

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 2–10 · Men only
Healthy volunteers
Accepted
Study type
Interventional · Not applicable
Target enrollment
50
US sites recruiting
1
Study start
March 4, 2026
Record last updated
April 8, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study 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.

Being studied: Descending stair walk

Conditions: Duchenne Muscular Dystrophy (DMD); Becker's Muscular Dystrophy (BMD)

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

DMD/BMD Subject Inclusion/Exclusion Criteria

Inclusion Criteria

  • 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

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

  • 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

Recruiting US locations

  • Children's Hospital of Philadelphia

    Philadelphia, PA 19104

    Site contact: Calliope O'Donnell, MS · 267-426-7786 · odonnellcs@chop.edu

Study contacts (from the official record)

  • Calliope O'Donnell, MS · 267-426-7786 · odonnellcs@chop.edu
  • Julianne Larosa, MPH · 267-426-7875 · Larosaj1@chop.edu
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Urinary Titin Biomarker in DMD (NCT07332013) | Bored Studies