Duchenne Electronic Health Record Study

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
The Duchenne Registry
Study ID
NCT07609394
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Observational study with patients who may be treated with various disease-modifying therapies — OTHER
    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.

Study Details

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 Dates

Start date
Dec 1, 2022
Status verified
May 2026
Primary completion
Dec 31, 2035
Completion
Dec 31, 2072

Study Design

Enrollment
2,500 participants (estimated)

Primary Outcome Measure

Progressive Muscle Weakness [ Time Frame: Date 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. ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202-
UC Davis HealthSacramentoCalifornia95817-
Children's Hospital ColoradoAuroraColorado80045-
Yale Children's HospitalNew HavenConnecticut06511-
Children's National Medical CenterWashington D.C.District of Columbia20010-
University of Iowa Health CareIowa CityIowa52242-
Duke University Medical CenterDurhamNorth Carolina27710-
UT Southwestern Medical CenterDallasTexas75390-
Primary Children's HospitalSalt Lake CityUtah84113-
University of Utah HealthSalt Lake CityUtah84132-

Find similar trials in Little Rock, AR

By research site
Arkansas Children's Hospital· Little Rock, ARUC Davis Health· Sacramento, CAChildren's Hospital Colorado· Aurora, COYale Children's Hospital· New Haven, CTChildren's National Medical Center· Washington D.C., DCUniversity of Iowa Health Care· Iowa City, IA

Related Studies