Duchenne Muscular Dystrophy/Becker Muscular Dystrophy

Data Standards

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

•         CDEs — Classified as Core, Supplemental–Highly Recommended, Supplemental, or Exploratory

•         CRF Modules — Template forms that logically organize CDEs for data collection

•         Guidance Documents — Provide further information about the CDEs

•         Instrument Notice of Copyright (NOC) Documents Include pertinent information on recommended instruments

Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Start-up Resource Listing: All Core and Supplemental–Highly Recommended CDEs recommended for Duchenne Muscular Dystrophy/Becker Muscular Dystrophy study start-up

Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Highlight Summary: Overview of all Duchenne Muscular Dystrophy/Becker Muscular Dystrophy-specific CDE recommendations as they appear on the website

Click Expand All to view the CDEs associated with the CRF modules, organized by domain and subdomain.

Form Search Instructions - Please Read Before Searching

The Form Search is designed to filter the CRF Modules, Guidance Documents, and NOCs on this page by Search Term, Domain, Subdomain, and/or Copyright Status. The Copyright field is designed to filter between forms that are freely available online and forms that are copyrighted or trademarked. Please note that regardless of copyright/trademark status, these instruments are not available on this website. All recommended instruments, regardless of copyright status, have informational documents posted on the NINDS CDE website which provide details concerning availability.

Helpful Hints:

Once you have completed your search, the search results will need to be expanded by clicking the Expand All button to view results.

The default setting for each field is ‘Any’.

When searching on the Traumatic Brain Injury or Sports Related Concussion disorder pages selecting a Timeframe or Sub-Disease will return more specific results.

For best results, click the Clear button between searches.

Please contact NINDS CDE team (NINDSCDE@emmes.com) in case you encounter any search difficulties.

Click here for additional instructions

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

Overview

The first set of Common Data Elements (CDEs) for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy was developed in 2012.

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. 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 formed prior to the Neuromuscular Diseases CDE Working Group was focused on developing Congenital Muscular Dystrophy (CMD) CDEs for a natural history study. These CDEs are available as a zip file on the CMD Overview page.

Roster

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

Publications

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