Duchenne Muscular Dystrophy/Becker Muscular Dystrophy

Data Standards

The National Institute of Neurological Disorders and Stroke (NINDS) and other Federal agencies and international organizations have the common mission of developing data standards for clinical research. Through the efforts of subject-specific working groups, topic-driven data elements have been created. The first set of Common Data Elements (CDEs) for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy was developed in 2012. The Core data elements to be used by an investigator when beginning a research study in this disease/disorder are listed in the Start-up Resource Listing.

Many of the CDEs will overlap across study types, which allows for comparisons and meta-analysis across studies. Consistency of the data elements and the CDE formats is kept in order to ensure the ability to transfer critical medical information electronically from one center to another. This consistency also allows for continuity across different disease areas. The goals of the NINDS CDE initiative are to increase the efficiency and effectiveness of clinical research studies and clinical treatment, increase data quality, facilitate data sharing, and help educate new clinical investigators.

Organized by domains and sub-domains, often used in clinical studies, data standards include:

Please view additional tabs for more information about the development of these CDEs. An overview of all Duchenne Muscular Dystrophy (DMD)/Becker Muscular Dystrophy (BMD) CDE recommendations can be found in the DMD CDE Highlight Summary document. For your reference, a zip file containing all the current Duchenne Muscular Dystrophy (DMD)/Becker Muscular Dystrophy (BMD) CDE template CRF modules can be downloaded below.

Participant Characteristics
Demographics
CRF Module/Guideline CDEs
Demographics CDE Details
General Core CDE Details
Participant History and Family History
General Health History
CRF Module/Guideline CDEs
Family History CDE Details
Medical History CDE Details
Surgical History CDE Details
Disease/Injury Related Events
History of Disease/Injury Event
CRF Module/Guideline CDEs
Medical History of DMD CDE Details
Treatment/Intervention Data
Devices
CRF Module/Guideline CDEs
External Devices and Other Treatments CDE Details
Therapies
CRF Module/Guideline CDEs
GI Therapies CDE Details
Respiratory Interventions CDE Details
Drugs
CRF Module/Guideline CDEs
Prior and Current Medications CDE Details

Overview

In order to create a comprehensive set of CDEs for Neuromuscular Diseases, the NINDS formed the following unique Working Groups: Neuromuscular Diseases (NMD), Myasthenia Gravis (MG), Spinal Muscular Atrophy (SMA), and Duchenne/Becker Muscular Dystrophy (DMD/BMD).

The NMD CDE Working Group’s goal was to identify elements that would be useful across multiple types of NMD Clinical Studies. In order to achieve this objective, the NMD Working Group was divided into two focus areas – Adult studies and Pediatric studies. In both the Adult and Pediatric areas, subgroups were formed to focus on defining data elements in the domains of:

  • Biomarkers/Genetics/Imaging
  • Cardiac/Pulmonary
  • Clinical Outcomes
  • Cognitive/Behavioral/Psychological Assessments
  • Demographics

While the Adult and Pediatric Working Groups initially worked separately, their age-specific NMD recommendations were later combined to form Version 1.0 of the NMD CDEs.

Additionally, the NINDS formed Working Groups to develop recommendations specific for Spinal Muscular Atrophy (SMA), Myasthenia Gravis (MG), and Duchenne and Becker Muscular Dystrophy (DMD/BMD). These three Working Groups were responsible for reviewing the NMD CDEs and forming disease-specific recommendations to supplement the overarching Neuromuscular Diseases CDEs.

Another Working Group was form prior to the Neuromuscular Diseases CDE Working Group and was focused on developing Congenital Muscular Dystrophy (CMD) CDEs for a natural history study. These CDEs are not incorporated with the NMD Diseases CDEs but are available as a zip file on the NMD data standards page.

History and Acknowledgements

The National Institute of Neurological Disorders and Stroke (NINDS) has formed an Oversight Committee to direct the updating and further development of Neuromuscular Diseases CDEs.

The Neuromuscular Diseases CDE Committee was formed first and focused on a set of general CDE recommendations to be utilized in clinical research across all neuromuscular diseases. Additional CDE Working Groups were subsequently formed to develop disease-specific recommendations for:

All three Working Groups (MG, SMA, and DMD/BMD) were charged with reviewing the combined Adult and Pediatric NMD CDEs to determine the additional elements needed for an MG, SMA, or DMD/BMD specific study.

All Working Group members actively developed the CDEs for their specific disease areas/subgroups and had an opportunity to review and comment on the draft recommendations internally prior to public review and posting. The NMD, MG, SMA, and DMD/BMD CDE Working Groups are supported by the NINDS CDE Team. The complete rosters for each Working Group and the rosters by NMD Subgroup are shown below. The Neuromuscular Diseases CDE Oversight Committee was created in 2013.

Complete Adult NMD Working Group Roster

Complete Pediatric NMD Working Group Roster

Complete Myasthenia Gravis (MG) Working Group Roster

Complete Spinal Muscular Atrophy (SMA) Working Group Roster

Complete Duchenne and Becker Muscular Dystrophy Working Group Roster

Complete NMD Oversight Committee Roster

 

Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (DMD/BMD) Working Group

  • Katherine Mathews, MD
    University of Iowa Children's Hospital, Iowa City, Iowa
    Chair
  • Katie Bushby, MD
    Newcastle University, Newcastle upon Tyne, United Kingdom
  • Kevin Flanigan, MD
    Nationwide Children's Hospital, Columbus, Ohio
  • Julaine M. Florence, PT, MHS, DPT
    Washington University School of Medicine, St. Louis, Missouri
  • Edward Kaye, MD
    SAREPTA Therapeutics, Cambridge, Massachusetts
  • Shree Pandya, PT, DPT, MS
    University of Rochester, Rochester, New York
  • Elise Townsend, DPT, PhD, PCS
    Massachusetts General Hospital, Boston, Massachusetts

NINDS CDE Team

  • Robin Conwit, MD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Elizabeth McNeil, MD, MSc
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Glen Nuckolls, PhD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Joanne Odenkirchen, MPH (Until 2016)
    NINDS CDE Project Officer, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Sherita Ala'I, MS (Until 2018)
    The Emmes Corporation
  • Marybeth Montoro, MPH, CPH (Until 2015)
    The Emmes Corporation
  • Lisa Hunegs, MSW, MPH (Until 2012)
    KAI Research, Inc. (An Altarum Company), Rockville, Maryland
  • Kristy Miller, MPH (Until 2012)
    KAI Research, Inc. (An Altarum Company), Rockville, Maryland
  • Christina You, MSPH (Until 2012)
    KAI Research, Inc. (An Altarum Company), Rockville, Maryland

References

A publication related to the NMD CDE initiative is in development. The citation for this publication will be included on this site as soon as it is available.

Updates

Please see attached revision history document. Please contact NINDSCDE@emmes.com if you require further information or have any questions about the revision history.

Duchenne and Becker Muscular Dystrophy CDE Revision History