Congenital Muscular Dystrophy

Data Standards

The National Institute of Neurological Disorders and Stroke (NINDS), 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 Congenital Muscular Dystrophy were developed in 2014-2015. The Core CDEs 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-analyses 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:

An overview of all Congenital Muscular Dystrophy CDE recommendations can be found in the Congenital Muscular Dystrophy CDE Highlight Summary document. For your reference, a zip file containing all the current Congenital Muscular Dystrophy CDE template CRF modules can be downloaded below.

The outline that follows includes all the CDEs associated with the CRF modules, organized by domain and sub-domain.

Overview of Working Group Recommendations
Summary Documents
CRF Module/Guideline CDEs
Summary of Recommendations for CMD N/A
Participant Characteristics
Social Status
CRF Module/Guideline CDEs
Social Status CDE Details
Demographics
CRF Module/Guideline CDEs
General Core CDE Details
Demographics CDE Details
Treatment/Intervention Data
Drugs
CRF Module/Guideline CDEs
Prior and Concomitant Medications CDE Details
Therapies
CRF Module/Guideline CDEs
Respiratory Interventions CDE Details
Outcomes and End Points
Performance Measures
CRF Module/Guideline CDEs
Motor Function Measure (MFM) N/A
Quality of Life/Patient Reported Outcomes
CRF Module/Guideline CDEs
Patient-Reported Outcomes Measurement Information System (PROMIS) N/A
Quality of Life in Neurological Disorders (Neuro-QOL) N/A

Overview

To develop the Congenital Muscular Dystrophy (CMD) CDEs, the CMD CDE Working Group met from April 2014 to October 2014. The recommendations were posted for public comment in November 2014 and version 1.0 was released at the end of February 2015.

Other CMD CDE development:

In 2010, the CMD CDE Project developed CDEs created in collaboration between the NINDS and Cure CMD. These CDEs are tailored specifically for a CMD natural history study and may be reused as appropriate.

Download Congenital Muscular Dystrophy (CMD) Natural History Study CDE Package

History and Acknowledgements

The Congenital Muscular Dystrophy (CMD) CDE Working Group has developed the CDEs for their recommendations. The CMD CDE Working Group is supported by the NINDS CDE Team. The Working Group began meeting by teleconference in May 2014 and held calls every four to six weeks to define the CDEs for their domains and to recommend standardized, validated instruments for CMD research.

Complete Congenital Muscular Dystrophy (CMD) CDE Roster

The complete CMD CDE Working Group roster is shown below.

CMD Working Group

  • Angela Giacoletti Argento, PhD
    University of Michigan Hospital and Health Systems, Ann Arbor, Michigan
  • Carsten BÖnnemann, MD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Jim J. Collins, MD, PhD
    Cincinnati Children's Medical Center, Cincinnati, Ohio
  • James Dowling, MD, PhD
    The Hospital for Sick Children, Toronto, Canada
  • Susan T. Iannaccone, MD, FAAN
    University of Texas Southwestern Medical Center, Dallas, Texas
  • Chamindra Konersman, MD
    Medical College of Wisconsin, Milwaukee, Wisconsin
  • Michael W. Lawlor, MD, PhD
    Medical College of Wisconsin, Milwaukee, Wisconsin
  • Kathy Mathews, MD
    University of Iowa Children's Hospital, Iowa City, Iowa
  • Francesco Muntoni, MD
    University College London, Institute of Child Health-Dubowitz, London, United Kingdom
  • Susan E. Sparks, MD, PhD
    Levine Children's Hospital, Charlotte, North Carolina

NIH Coordinating 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
  • Yaffa Rubinstein, PhD
    National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Bethesda, Maryland
  • Tiina K. Urv, PhD
    National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH), Bethesda, Maryland

NINDS CDE Team

  • Sherita Ala'i, MS (Until 2018)
    The Emmes Corporation
  • Joy Esterlitz, MS
    The Emmes Corporation
  • Robin Feldman, BS, MBA
    The Emmes Corporation
  • Muniza Sheikh, MS, MBA
    The Emmes Corporation

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.

Congenital Muscular Dystrophy CDE Revision History Document