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.
Highlights
The dystrophinopathies, Duchenne muscular dystrophy (DMD) and allelic Becker muscular dystrophy (BMD), are X-linked recessive disorders caused by mutations in the DMD gene that result in deficient dystrophin production [1]
17.9% of 358 individuals ascertained by the MD STARnet were identified as participating in a clinical trial (Table 1)
Corticosteroids, tadalafil, and ataluren (PTC124) were the most common clinical trial medications recorded. The age of those individuals in 2015 who had participated in a clinical trial during 2000–2015 tended to cluster between 7 and 12 years (Figure 3) and most clinical trial medications were identified as having started during 2013–2015 (Figure 4)
Summary
The dystrophinopathies, Duchenne muscular dystrophy (DMD) and allelic Becker muscular dystrophy (BMD), are X-linked recessive disorders caused by mutations in the DMD gene that result in deficient dystrophin production [1]. DMD is associated with mutations that disrupt the reading frame or lead to a premature stop, resulting in a complete absence of dystrophin in muscle. BMD includes a wide spectrum of severity and is historically distinguished from DMD by having loss of independent ambulation after age 16 years. Therapeutic trials are critical to improving outcomes for individuals diagnosed with Duchenne muscular dystrophy (DMD). Clinical trial participation and individual-level clinical and sociodemographic characteristics were obtained from medical records for the 2000–2015 calendar years. Results: Clinical trial participation was identified among 17.9% (MD STARnet site: 3.7–27.3%) of 358 individuals with DMD. Fewer non-Hispanic blacks or Hispanics than non-Hispanic whites participated in clinical trials. 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
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