Urinary Titin Biomarker in DMD

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT07332013
Status
Recruiting
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Conditions

  • Becker's Muscular Dystrophy (BMD)
  • Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

Sex
MALE
Age
2 Years - 10 Years
Healthy Volunteers
Accepted

Interventions

  • Descending stair walk — OTHER
    Subjects will participate in a brief on-site, descending stair walk. Subjects will walk down stairs, up to a maximum 2 floors, under the supervision of a physical therapist or study team member.

Study Details

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.

Key Dates

Start date
Mar 4, 2026
Status verified
Apr 2026
Primary completion
Dec 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
50 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE

Arms

  • Experimental: Experimental

Primary Outcome Measure

Change in urinary NTFT (titin) after structured activity [ Time Frame: Day 1, 6-12 months , 12-18 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Calliope O'Donnell, MS
[email protected]
267-426-7786

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By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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