A Registry-Based Perspective of Interventional Clinical Trials for Duchenne Muscular Dystrophy

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Background and objective Duchenne muscular dystrophy (DMD) is a severe, progressive, X-linked genetic disorder caused by pathogenic variants in the dystrophin gene, leading to progressive muscle weakness, loss of ambulation, and premature mortality. Clinical trials are essential for advancing evidence-based management of DMD; however, the overall patterns of study design, intervention strategies, funding, and results reporting remain incompletely characterized. This study aimed to characterize interventional DMD clinical trials registered in ClinicalTrials.gov as of May 25, 2026, and to assess patterns in study design, intervention type, trial phase, funding, results reporting, and trial start era. Methods A retrospective registry-based descriptive study was conducted using ClinicalTrials.gov. DMD-related records were identified using the free-text terms "Duchenne muscular dystrophy," "Duchenne," and "DMD"; the search was not restricted by trial start year. Eligible records were interventional trials with a DMD-specific objective. Trial-level eligibility characteristics, study design, intervention type, trial phase, funding source, results posting, and start year were summarized. Era-wise comparisons were performed using the Fisher-Freeman-Halton exact test. Results A total of 232 interventional DMD trial records were included. Male-only eligibility was recorded for 198/232 trials (85.3%), and treatment was the primary purpose in 179/232 (77.2%). Drug interventions were the most frequent coded intervention category (101/232; 43.5%), Phase 2 was the largest phase category (58/232; 25.0%), and industry was the most common funder class (118/232; 50.9%). Results were posted on ClinicalTrials.gov for 76/232 trials (32.8%). The proportion of drug-intervention trials differed across start-year eras (p = 0.002). Biological interventions showed numerically greater representation in the most recent era, but the era-wise difference was not statistically significant (p = 0.420); industry sponsorship, Phase 3/4 status, randomization, and masking also did not differ significantly across eras. Conclusions ClinicalTrials.gov records show a predominantly treatment-oriented DMD research profile, with substantial industry involvement and frequent small-cohort studies. Therapeutic modalities are diverse, with biological and genetic approaches representing an important share of recent registered activity; however, most temporal comparisons of trial-design characteristics were not statistically significant. Limited registry results posting remains an important gap.

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