Urine titin test for Duchenne and Becker muscular dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The study measures the urinary N-terminal fragment of titin (NTFT) before and after a controlled activity (descending two flights of stairs) and during seven days of home monitoring to link biomarker levels to activity. Participants complete standard neuromuscular tests (North Star Ambulatory Assessment and timed function tests) and wear an activity monitor during clinic visits and at home. Urine is collected pre- and post-exercise at clinic visits and three times daily during the home monitoring period, with optional exploratory blood sampling if a clinical blood draw occurs. Investigators will correlate urinary NTFT with activity intensity and clinical severity to determine whether NTFT is a sensitive, noninvasive marker of systemic muscle injury.

Why it matters

Who can join: the full rules

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

Where it's running

• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: Sabrina Yum, MD — Children's Hospital of Philadelphia
• Study coordinator: Calliope O'Donnell, MS
• Email: odonnellcs@chop.edu
• Phone: 267-426-7786

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.