Duchenne Electronic Health Record Study

Recruiting now

Conditions studied: Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier

In brief

This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer. At select clinics across the United States, electronic health record (EHR) data from consented patients will be pushed into PPMD's Duchenne Outcomes Research Interchange (the Interchange), where the EHR data can be combined with patient-reported data from The Duchenne Registry. By combining this data in a central hub, we will gain a more complete picture of Duchenne and Becker muscular dystrophy, allowing researchers and clinicians to develop treatments faster and to improve and refine the standards of care for Duchenne and Becker. The ultimate goal is to optimize function, quality of life, and survival of Duchenne and Becker patients. EHR data collected will be fully identifiable retrospective data for core clinical data elements going back ten years (as available) from the date of consent; going back one year for retrospective clinical notes from the date of consent; and prospectively collecting both core clinical data elements and clinical notes. Information collected will align with the FHIR U.S. core data elements, also known as the Common Clinical Data Set. PPMD partnered with Prometheus Research (an IQVIA company), an industry leader in health data informatics, to launch both the EHR Study and the Interchange. All data is stored securely and in accordance with strict industry standards and patient privacy laws. Participation in the EHR data extraction is voluntary, and a patient can withdraw consent at any time.

Key facts

Study ID

NCT07609394

Run by

The Duchenne Registry

People needed

2500

Starts

2022-12-01

Expected to finish

2072-12-01

Last updated by the study team

2026-05-27

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Duchenne or Becker muscular dystrophy or female carrier
• Must be a patient at an institution that has an established EHR integration set up with PPMD's Interchange
• Must provide consent to have their EHR data pushed to the Interchange and linked to existing Registry data, if applicable

You may not qualify if…

• Individuals with other forms of muscular dystrophy
• Individuals who do not provide consent
• Individuals with Duchenne/Becker who have severe mobility/strength issues need to provide consent and participate with assistance from a caregiver. Adults with communication impairments and/or intellectual disabilities (considered the "decisionally impaired" group for purposes of this study) will be able to consent with the assistance of the adults who are designated Legally Authorized Representative (LAR). Without assistance, this group will be excluded from participation because the consent process.

Where it is running

• University of Utah Health — Salt Lake City, Utah, United States (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Yale Children's Hospital — New Haven, Connecticut, United States (enrolling)
• Children's National Medical Center — Washington D.C., District of Columbia, United States (enrolling)
• University of Iowa Health Care — Iowa City, Iowa, United States (enrolling)
• Duke University Medical Center — Durham, North Carolina, United States (enrolling)
• UT Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• Primary Children's Hospital — Salt Lake City, Utah, United States (enrolling)
• UC Davis Health — Sacramento, California, United States

Full record on ClinicalTrials.gov

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