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Duchenne Electronic Health Record Study

21 maj 2026 uppdaterad av: The Duchenne Registry

Duchenne Outcomes Research Interchange Data Enrichment Through EHR Extraction

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Studietyp

Observationell

Inskrivning (Beräknad)

2500

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studera Kontakt Backup

Studieorter

  • Förenta staterna
    • Arkansas
      • Little Rock, Arkansas, Förenta staterna, 72202
        • Rekrytering
        • Arkansas Children's Hospital
    • California
      • Sacramento, California, Förenta staterna, 95817
        • Har inte rekryterat ännu
        • UC Davis Health
    • Colorado
      • Aurora, Colorado, Förenta staterna, 80045
        • Rekrytering
        • Children's Hospital Colorado
    • Connecticut
      • New Haven, Connecticut, Förenta staterna, 06511
        • Rekrytering
        • Yale Children's Hospital
    • District of Columbia
      • Washington D.C., District of Columbia, Förenta staterna, 20010
        • Rekrytering
        • Children's National Medical Center
    • Iowa
      • Iowa City, Iowa, Förenta staterna, 52242
        • Rekrytering
        • University of Iowa Health Care
    • North Carolina
      • Durham, North Carolina, Förenta staterna, 27710
        • Rekrytering
        • Duke University Medical Center
    • Texas
      • Dallas, Texas, Förenta staterna, 75390
        • Rekrytering
        • UT Southwestern Medical Center
    • Utah
      • Salt Lake City, Utah, Förenta staterna, 84113
        • Rekrytering
        • Primary Children's Hospital
      • Salt Lake City, Utah, Förenta staterna, 84132
        • Rekrytering
        • University of Utah Health

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Barn
  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Testmetod

Icke-sannolikhetsprov

Studera befolkning

Individuals with dystrophinopathy (including Duchenne, Becker, and carriers) who are patients at a Certified Duchenne Care Center (CDCC) in the United States that has an established EHR integration with PPMD's Interchange.

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Progressive Muscle Weakness
Tidsram: 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.
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).
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.
Cardiac Function
Tidsram: Date 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.
Date 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.
Pulmonary Function
Tidsram: FVC 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.
FVC 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.
Bone Health
Tidsram: BMI, 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.
BMI, 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.

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

The Duchenne Registry

Samarbetspartners

Parent Project Muscular Dystrophy

Utredare

  • Huvudutredare: Ann Martin, MS, CGC, Parent Project Muscular Dystrophy
  • Huvudutredare: Eric Camino, PhD, Parent Project Muscular Dystrophy
  • Huvudutredare: Rachel Schrader, MS, APRN, CPNP-PC, Parent Project Muscular Dystrophy

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Användbara länkar

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

1 december 2022

Primärt slutförande (Beräknad)

1 december 2035

Avslutad studie (Beräknad)

1 december 2072

Studieregistreringsdatum

Först inskickad

19 mars 2026

Först inskickad som uppfyllde QC-kriterierna

21 maj 2026

Första postat (Faktisk)

27 maj 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

27 maj 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

21 maj 2026

Senast verifierad

1 maj 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • EHR-PPMD-2026

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

De-identified IDP may be shared with researchers following approval by the Duchenne Outcomes Research Interchange Steering Committee.

IPD-delning som stöder informationstyp

  • STUDY_PROTOCOL
  • ICF

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Nej

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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