Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
Year: 2021
Type: article
Study design: Observational Study
Abstract: Background: Therapeutic trials are critical to improving outcomes for individuals diagnosed with Duchenne muscular dystrophy (DMD). Understanding predictors of clinical trial participation could maximize enrollment. Methods: Data from six sites (Colorado, Iowa, Piedmont region North Carolina, South Carolina, Utah, and western New York) of the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) were analyzed. Clinical trial participation and individual-level clinical and sociodemographic characteristics were obtained from medical records for the 2000–2015 calendar years. County-level characteristics were determined from linkage of the most recent county of residence identified from medical records and publicly available federal datasets. Fisher’s exact and Wilcoxon two-sample tests were used with statistical significance set at one-sided p-value (<0.05) based on the hypothesis that nonparticipants had fewer resources. Results: Clinical trial participation was identified among 17.9% (MD STARnet site: 3.7–27.3%) of 358 individuals with DMD. Corticosteroids, tadalafil, and ataluren (PTC124) were the most common trial medications recorded. Fewer non-Hispanic blacks or Hispanics than non-Hispanic whites participated in clinical trials. Trial participants tended to reside in counties with lower percentages of non-Hispanic blacks. Conclusion: Understanding characteristics associated with clinical trial participation is critical for identifying participation barriers and generalizability of trial results. MD STARnet is uniquely able to track clinical trial participation through surveillance and describe patterns of participation.
Source: Children
Institutions: University of Iowa, Virginia Commonwealth University, Virginia Commonwealth University Medical Center, Centers for Disease Control and Prevention, National Center on Birth Defects and Developmental Disabilities, University of Utah, Roseman University of Health Sciences, University of Rochester Medicine, University of Rochester
Language: English
Topic: Ethics in Clinical Research
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $1,728
Awards: G1457604942, G254764699, G3604254668, G3889673609, G4449540638, COMPONENT A: IOWA MD STARNET CORE SITE, COMPONENT C - THE MUSCULAR DYSTROPHY SURVEILLANCE, TRACKING, AND RESEARCH NETWORK (MD STARNET) IN UTAH, COMPONENT C: IOWA MD STARNET SITE DATA DISSEMINATION, G7037899330, COMPONENT A - THE MUSCULAR DYSTROPHY SURVEILLANCE, TRACKING, AND RESEARCH NETWORK (MD STARNET) IN UTAH, G8401041936, G8633996454, G917963011
OpenAlex ID: W3202961278