Duchenne Electronic Health Record Study
This study is collecting long-term health information from people with Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, or who are symptomatic female carriers. It's an observational study, meaning researchers will gather information from your existing electronic health records (EHR) and combine it with information you might have already shared with The Duchenne Registry. This includes details about your muscle weakness, heart function, and lung function over many years. You can participate even if you are taking various treatments like corticosteroids, exon skipping therapy, or gene therapy, or therapies in other clinical trials. The goal is to create a more complete picture of these conditions to help doctors and researchers develop better treatments and improve care. The study aims to enroll 2500 participants, but its current recruitment status is unclear.
- Study design
- This is an observational study that aims to enroll 2500 participants. It collects data from electronic health records and combines it with patient-reported data.
- What's involved
- You would need to be a patient at a clinic that has an established electronic health record integration with PPMD's Interchange and provide consent for your EHR data to be shared. This data will be collected over an anticipated average of 20 years.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your health data, including muscle weakness, cardiac function, and pulmonary function, will be followed for an anticipated average of 20 years.
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Duchenne Electronic Health Record Study
At a glance
Conditions
NCT07609394
Where you'd take part
This study runs at 10 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Arkansas Children's Hospital
Little Rock, Arkansasno site contact published
Recruiting
Children's Hospital Colorado
Aurora, Coloradono site contact published
Recruiting
Children's National Medical Center
Washington D.C., District of Columbiano site contact published
Recruiting
Duke University Medical Center
Durham, North Carolinano site contact published
Recruiting
Primary Children's Hospital
Salt Lake City, Utahno site contact published
Recruiting
UC Davis Health
Sacramento, Californiano site contact published
Not yet recruiting
University of Iowa Health Care
Iowa City, Iowano site contact published
Recruiting
University of Utah Health
Salt Lake City, Utahno site contact published
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Ann Martin, MS, CGC · PRINCIPAL_INVESTIGATOR · Parent Project Muscular Dystrophy
- Eric Camino, PhD · PRINCIPAL_INVESTIGATOR · Parent Project Muscular Dystrophy
- Rachel Schrader, MS, APRN, CPNP-PC · PRINCIPAL_INVESTIGATOR · Parent Project Muscular Dystrophy
Who to contact
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Inclusion
Exclusion
What this trial measures
- Progressive Muscle WeaknessDate of initiation of corticosteroids and date of first wheelchair/DME order; Steroid use recorded at baseline (day 1) and each annual follow-up visit (until patient is no longer seen at institution or withdraws consent), anticipated average of 20 years.
Characterize progressive muscle weakness in dystrophinopathy patients over time by measuring 1) age at start of corticosteroids (age at first prescription); 2) corticosteroid use including name, dose, regimen; and 3) dependence on wheelchair or age at fulltime wheelchair use (date of wheelchair/DME order).
- Cardiac FunctionDate of first echo, cardiac MRI, and EKG and all follow-up scans recorded at each annual visit (until patient is no longer seen at institution or withdraws consent), anticipated average of 20 years; Date of first ACE inhibitor or ARB prescription.
Characterize cardiac standard of care and cardiac function in dystrophinopathy patients by measuring 1) age at first echocardiogram, cardiac MRI, and EKG; 2) age at first ACE inhibitor or ARB prescription; and 3) recording LVEF on echocardiogram and cardiac MRI throughout study.
- Pulmonary FunctionFVC and PCF recorded at baseline (day 1) and at each annual follow-up visit (until patient is no longer seen at institution or withdraws consent), anticipated average of 20 years.
Characterize pulmonary standard of care and pulmonary function in dystrophinopathy patients by measuring spirometry results including 1) forced vital capacity (FVC), % predicted; and 2) peak cough flow (PCF) in L/min.
- Bone HealthBMI, Xray of spine and DEXA scan recorded at baseline (day 1) and at each annual follow up visit (until patient is no longer seen at institution or withdraws consent), anticipated average of 20 years; Date of first bisphosphonates prescription.
Characterize orthopedic standard of care and bone health in dystrophinopathy patients by measuring 1) date of first Xray of spine and DEXA scan; 2) age at first bisphosphonates prescription; and 3) recording BMI throughout study.