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Urinary Titin Biomarker in DMD

NCT07332013 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-04-08

No results posted yet for this study

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.

Conditions

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

Interventions

OTHER

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.

Sponsors & Collaborators

Principal Investigators

  • Sabrina Yum, MD · Children's Hospital of Philadelphia

  • Benjamin Kozyak, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
10 Years
Sex
MALE
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2026-03-04
Primary Completion
2029-12-31
Completion
2029-12-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07332013 on ClinicalTrials.gov