Clinical Research Directory
Browse clinical research sites, groups, and studies.
Urinary Titin Biomarker in DMD
Sponsor: Children's Hospital of Philadelphia
Summary
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.
Official title: Non-invasive Evaluation of Urinary Titin as an IND-enabling Biomarker for Use in Duchenne Muscular Dystrophy (DMD) Clinical Trials
Key Details
Gender
MALE
Age Range
2 Years - 10 Years
Study Type
INTERVENTIONAL
Enrollment
50
Start Date
2026-03-04
Completion Date
2029-12
Last Updated
2026-04-08
Healthy Volunteers
Yes
Interventions
Descending stair walk
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.
Locations (1)
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States