Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

Katherine D Mathews; Kristin M Conway; Amber M Gedlinske; Nicholas Johnson; Natalie Street; Russell J Butterfield; Man Hung; Emma Ciafaloni; Paul A Romitti

doi:10.3390/children8100835

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Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

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Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)

Katherine D Mathews, Kristin M Conway, Amber M Gedlinske, Nicholas Johnson, Natalie Street, Russell J Butterfield, Man Hung, Emma Ciafaloni and Paul A Romitti

Children (Basel), Vol.8(10), p.835

09/23/2021

DOI: 10.3390/children8100835

PMCID: PMC8534386

PMID: 34682100

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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.

Details

Title: Subtitle: Characteristics of Clinical Trial Participants with Duchenne Muscular Dystrophy: Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
Creators: Katherine D Mathews
Kristin M Conway
Amber M Gedlinske
Nicholas Johnson
Natalie Street
Russell J Butterfield
Man Hung
Emma Ciafaloni
Paul A Romitti
Resource Type: Journal article
Publication Details: Children (Basel), Vol.8(10), p.835
DOI: 10.3390/children8100835
PMID: 34682100
PMCID: PMC8534386
NLM abbreviation: Children (Basel)
ISSN: 2227-9067
eISSN: 2227-9067
Grant note: DOI: 10.13039/100000030, name: Centers for Disease Control and Prevention, award: DD001126, DD001119, DD001123, DD001116, DD001117, DD001108, DD001120, DD001054
Language: English
Date published: 09/23/2021
Academic Unit: Neurology; Stead Family Department of Pediatrics; Epidemiology; Iowa Neuroscience Institute; Biostatistics; Neurology (Pediatrics); Internal Medicine
Record Identifier: 9984187041002771

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