Urinary Titin Biomarker in DMD

Plain-language summary

This study evaluates a urinary biomarker called titin in children with Duchenne or Becker muscular dystrophy (DMD/BMD), using a descending stair walk as part of the assessment. It enrolls ambulatory boys with a genetically confirmed DMD/BMD diagnosis, along with healthy children for comparison. This is a study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

Children's Hospital of Philadelphia

Intervention

Descending stair walk

Start date

3/4/2026

Estimated completion

12/1/2029

Countries

United States

Related genes

Locations

• Philadelphia, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

DMD/BMD Subject Inclusion/Exclusion Criteria

Inclusion Criteria:

1. Ambulatory at screening
2. Genetically confirmed diagnosis of DMD/BMD
3. 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
4. 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:

1. Healthy children without DMD, BMD, or other significant chronic medical disease
2. Ambulatory at Screening, defined as able to walk independently without assistive devices
3. 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.
4. 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