Duchenne/Becker Muscular Dystrophy EHR Data Linkage

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational project collects retrospective and prospective electronic health record data from consented patients at select US clinics and links it to data in The Duchenne Registry via PPMD's Interchange. The goal is to build a centralized dataset that combines clinical information with patient-reported outcomes to better understand disease progression and care practices. Researchers and clinicians may use the linked data to identify patterns and inform faster development of treatments and standards of care. The study involves various disease-modifying therapies as part of routine care and uses identifiable data for up to ten years prior to consent, subject to consent and data sharing arrangements. Participation requires consent, consented data transfer, and contribution from clinicians with established EHR integration capabilities.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• UC Davis Health
  Sacramento, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Yale Children's Hospital
  New Haven, Connecticut, United States Recruiting
• Children's National Medical Center
  Washington D.C., District of Columbia, United States Recruiting
• University of Iowa Health Care
  Iowa City, Iowa, United States Recruiting

Study contacts

• Principal investigator: Ann Martin, MS, CGC — Parent Project Muscular Dystrophy
• Study coordinator: Megan Freed, MPH
• Email: megan@parentprojectmd.org
• Phone: 800-714-5437

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Duchenne/Becker Muscular Dystrophy Registry for patients and carriers

  This registry tests whether patients with Duchenne or Becker muscular dystrophy, or symptomatic female carriers, can be enrolled and followed across specialist centers to improve data sharing and care.

  Recruiting at 19 sites University Hospital Heidelberg · Graz and 29 other locations · NCT07402122

• An online registry for Duchenne and Becker muscular dystrophy patients

  This study is creating an online registry to help people with Duchenne and Becker muscular dystrophy connect with clinical trials and find helpful information about their condition.

  Recruiting The Duchenne Registry · Washington D.C., District of Columbia · NCT02069756

• Modeling heart failure in Duchenne and Becker muscular dystrophy to find surrogate heart measures

  This study will look at heart disease in people with Duchenne/Becker muscular dystrophy and carriers to see if MRI-based heart measures can predict death or major heart problems.

  Recruiting at 9 sites Vanderbilt University Medical Center · Sacramento, California and 8 other locations · NCT07674758

• DMD gene changes and heart function in boys and young men with dystrophinopathies

  This study will test whether specific DMD gene changes and routine heart tests (blood biomarkers, ECG, and echocardiography) can help identify early heart problems in boys and young men aged 2–24 with genetically confirmed dystrophinopathies.

  Recruiting Aristotle University Of Thessaloniki · Thessaloniki · NCT07515235

• How chronic pain changes over time in kids with Duchenne Muscular Dystrophy

  This study is following children and teens with Duchenne Muscular Dystrophy for 6 months to see how their chronic pain changes and how it affects daily life.

  Recruiting Antalya Training and Research Hospital · Antalya, Antalya · NCT07892196

• Bone health in adults with Duchenne muscular dystrophy: osteoporosis, fractures, and treatment

  This study looks at how common bone problems and fractures are in adults with Duchenne muscular dystrophy and what factors from childhood might affect bone health later on.

  Recruiting at 15 sites Istituto Giannina Gaslini · Bologna and 14 other locations · NCT07890883

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.