A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children

Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age1-18
SponsorWest China Second University Hospital

About this trial

This is a prospective, multicenter, longitudinal observational cohort study aimed at understanding the progression of Duchenne Muscular Dystrophy (DMD). The primary objective is to identify and integrate key biomarkers from multiple sources-including motor function assessments, body composition (muscle and fat distribution), clinical laboratory tests, and cardiopulmonary imaging-to delineate comprehensive disease trajectories. By analyzing how these factors change over time in a large cohort, the study seeks to develop a robust model that can identify patterns of disease progression. The ultimate goal is to generate evidence that may aid in forecasting individual patient outcomes and inform the future development of personalized rehabilitation and therapeutic strategies.

Eligibility criteria

Qualifiers

Male participants with genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD)

Age range: 1 to 18 years old (adjust to your actual age limit)

Ability to complete study assessments and follow-up visits

Participants or legal guardians provide written informed consent

Disqualifiers

Participants with other neuromuscular disorders that may confound natural history data

Participation in another interventional clinical trial that could affect disease progression

Severe comorbidities that prevent completion of study assessments

Inability to provide informed consent or comply with study procedures

Trial design

Treatments tested in this trial

  • No interventions are applied; participants are followed for natural history observation only

Treatment groups

500 Participants
are divided into 1 treatment group