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Duchenne Electronic Health Record Study

NCT07609394 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 2500

Last updated 2026-05-27

No results posted yet for this study

Summary

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.

Conditions

  • Duchenne Muscular Dystrophy (DMD)
  • Becker Muscular Dystrophy
  • Dystrophinopathy
  • Dystrophinopathy Symptomatic Female Carrier

Interventions

OTHER

Observational study with patients who may be treated with various disease-modifying therapies

Patients may be on any combination of therapies to participate, including FDA-approved therapies (corticosteroids, exon skipping therapy, gene therapy) or therapies in clinical trial.

Sponsors & Collaborators

Principal Investigators

  • Ann Martin, MS, CGC · Parent Project Muscular Dystrophy

  • Eric Camino, PhD · Parent Project Muscular Dystrophy

  • Rachel Schrader, MS, APRN, CPNP-PC · Parent Project Muscular Dystrophy

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-01
Primary Completion
2035-12-31
Completion
2072-12-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07609394 on ClinicalTrials.gov