Urinary Titin Biomarker Study for DMD/BMD
This study is looking at a new way to measure muscle damage in Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) using a biomarker (a measurable indicator of a biological state) found in urine. Researchers believe that levels of urinary N-terminal fragment of titin (NTFT) could show how much muscle injury is happening and how well treatments are working. The study will involve 50 boys, aged 2 to 10, who can walk. Participants will do a short stair walk and have their urine collected before and after. They will also wear an activity monitor and collect urine samples at home over several days. The goal is to see if NTFT levels in urine change with activity and reflect the severity of the disease.
- Study design
- This is an interventional study with a planned enrollment of 50 participants. The study aims to measure changes in urinary NTFT after structured activity and during unstructured activity at home.
- What's involved
- You would attend 3 regular clinical visits where you will perform a descending stair walk and have urine collected. You will also wear an activity monitor and collect urine samples at home for 7 days after the first two visits.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 12 to 18 months, with measurements taken at Day 1, 6-12 months, and 12-18 months.
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Urinary Titin Biomarker in DMD
At a glance
Conditions
NCT07332013
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvaniastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Sabrina Yum, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
- Benjamin Kozyak, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
Who to contact
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Exclusion
What this trial measures
- Change in urinary NTFT (titin) after structured activityDay 1, 6-12 months , 12-18 months
Change in urinary NTFT concentration will be measured before and after clinical visits, during which subjects will complete standard of care physical therapy assessments and perform a two-flight stair descent (if able).
- Urinary NTFT (titin) relative during unstructured activity in home environmentDay 1, 6-12 months, 12- 18 months
Change in urinary NTFT (titin) concentration over 1 week in response to unstructured activity at home and will be measured after each scheduled visit. This period of home NTFT monitoring will occur at the same time as activity monitoring described in outcome measure #3.
- Unstructured activity level as assessed by wearable activity deviceDay 1, 6-12 months, 12-18 months
Subjects' daily movement and activity levels will be measured continuously over 1 week at home using a pair of wearable accelerometry sensors. This will be completed after each scheduled clinical visit. This period of home activity monitoring will occur in conjunction with NTFT monitoring as described in outcome measure #2.