Inequitable racial and ethnic representation in Duchenne Muscular Dystrophy Clinical Trials

Objective: We investigated racial and ethnic distribution of participants in Duchenne Muscular Dystrophy (DMD) Phase II and III clinical trials. Methods: A total of 36 DMD Phase II and III clinical trials were analyzed for racial and ethnicity information. Publicly available demographic information was collected from DMD Phase II and III clinical trials registered between 2005 to 2018 from the Clinical Trials database (clinicaltrials.gov). Clinical trial participation was also analyzed based on geographic location (international versus United States) and funding source (industry versus academia). Results: White participants accounted for 84% of study participants in DMD Phase II and Phase III clinical trials in both multinational studies and within continental United States. Among the 36 trials, 22% (8/36) did not report racial data and 44% (16/36) did not report ethnicity. Most DMD Phase II and III clinical trials were funded by industry (89%) compared to the National Institutes of Health (NIH) (3%), and other sources (8%). Interpretation: White participants are most represented in DMD Phase II and III clinical trials. The documentation of racial and ethnic information in DMD clinical trials are insufficient. These data highlight the need for further approaches to diversify and include equitable representation in DMD clinical trials.

Publication History

Received: 07 April 2025

Accepted after revision: 02 July 2025

Accepted Manuscript online:
03 July 2025