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GUP24005NCT07879989

Respiratory Progression in Adults With Duchenne Muscular Dystrophy.

StatusRecruiting
PhaseNot specified
Primary completion (estimated)
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Record History

All 1 version: 1 with changes below.

  1. Study First Submitted

Endpoints

Primary

Characterization of Respiratory Progression in Adults with Duchenne Muscular Dystrophy
Timeframe: 36 months; retrospective data collected from the earliest available assessment and prospective respiratory and motor assessments performed every 6 months.

Secondary (5)

Association of Motor, Respiratory, and Genetic Factors with Respiratory Failure
Validation of Diaphragm Ultrasound in Adults with Duchenne Muscular Dystrophy
Development of a Composite Clinical and Genetic Severity Score
Identification of Genetic and Serum Biomarkers of Respiratory Disease Progression
Development of a Predictive Algorithm for Respiratory Disease Progression

Arms and interventions

Adults with Duchenne Muscular Dystrophy (DMD)

Adults aged 18 years or older with genetically confirmed Duchenne muscular dystrophy, not enrolled in interventional clinical trials, who provide informed consent. Clinical, motor, respiratory, and genetic data will be collected retrospectively and prospectively over 36 months. Assessments include spirometry, inspiratory pressure measurements, overnight respiratory and sleep studies, and, in a subgroup, diaphragm ultrasound. Blood samples will be analyzed to identify genetic modifiers and inflammatory biomarkers associated with respiratory disease progression. No experimental intervention is planned; the study is observational.

Sites

13 sites in 1 country