Clinical Trial Landscape of Duchenne Muscular Dystrophy in Europe: A 10-Year Retrospective Analysis

Background and Objectives: Duchenne muscular dystrophy (DMD) is a rare and progressive genetic disease with limited treatment options. Clinical trials remain essential for the development of new therapies and may provide patients with access to new potential treatments. This study aims to describe the clinical trial landscape for DMD therapies in the European Union and European Economic Area (EU/EEA) between 2016 and 2026. Materials and Methods: A retrospective descriptive analysis was conducted using publicly available data from EudraCT and the Clinical Trials Information System (CTIS). Interventional clinical trials investigating medicinal products for DMD were included if they were active between January 2016 and June 2026 and had at least one investigational site in an EU/EEA country. Results: The search identified 95 clinical trial records, of which 80 remained after deduplication, and 62 met the inclusion criteria. Phase III trials accounted for 37.1% (n = 23), followed by Phase II trials at 30.6% (n = 19). At the time of data extraction, 19 trials had been completed, 17 were ongoing, and 19 had been terminated prematurely. Five trials had been authorised but had not yet started recruitment, while two were temporarily halted due to safety concerns. Trial initiation varied between years, but no consistent trend was identified. Geographic participation was uneven, with the highest activity recorded in Italy, Spain, Belgium, and France. Conclusions: No consistent temporal trend in DMD trial initiation was observed during the study period. Most included trials were conducted in Phase II or III. However, the high number of terminated studies and the concentration of trial activity in a limited number of countries show that scientific, operational, and geographic challenges remain.