Congenital 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 (Instrument Notice of Copyright Information)

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

Congenital Muscular Dystrophy Highlight Summary: Overview of all Congenital 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.

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NIH Resources
NIH Resources
CRF Module/Guidance CDEs
National Institutes of Health (NIH) Toolbox N/A
Patient-Reported Outcomes Measurement Information System (PROMIS) N/A
Quality of Life in Neurological Disorders (Neuro-QOL) N/A
Overview of Working Group Recommendations
Summary Documents
CRF Module/Guidance CDEs
Summary of Recommendations for CMD N/A
Participant Characteristics
Social Status
CRF Module/Guidance CDEs
Social Status CDE Details
Demographics
CRF Module/Guidance CDEs
General Core CDE Details
Demographics CDE Details
Participant History and Family History
General Health History
CRF Module/Guidance CDEs
Family History CDE Details
Intake Medical History CDE Details
Interval Medical History CDE Details
Prenatal and Perinatal History CDE Details
Surgical History CDE Details
Assessments and Examinations
Imaging Diagnostics
CRF Module/Guidance CDEs
Brain Magnetic Resonance Imaging (MRI) CDE Details
Cardiac Magnetic Resonance Imaging (MRI) CDE Details
Diffusion Tensor Imaging (DTI) CDE Details
Dual-Energy X-Ray Absorptiometry (DEXA) CDE Details
Echocardiogram CDE Details
Muscle Imaging CDE Details
Physical/Neurological Examination
CRF Module/Guidance CDEs
Electrical Impedance Myography (EIM) N/A
Non-Imaging Diagnostics
CRF Module/Guidance CDEs
Electrophysiology CDE Details
Short Scalp Electroencephalography (EEG) CDE Details
Laboratory Tests and Biospecimens/Biomarkers
CRF Module/Guidance CDEs
Muscle Biopsies and Autopsy Tissue CDE Details
Peripheral Nerves - Biopsies and Autopsies CDE Details
Skin Biopsies for Qualification of Intraepidermal Nerve Fibers CDE Details
Treatment/Intervention Data
Drugs
CRF Module/Guidance CDEs
Prior and Concomitant Medications CDE Details
Therapies
CRF Module/Guidance CDEs
Respiratory Interventions CDE Details
Outcomes and End Points
Functional Outcomes
CRF Module/Guidance CDEs
10 Meter Timed Walk N/A
2 Minute Walk Test N/A
6 Minute Walk Test N/A
Alberta Infant Motor Scale N/A
Barthel Index N/A
Bayley Scales of Infant Development (Bayley III, BSID) N/A
Brooke Upper Extremity Scale N/A
Egen Klassifikation Scale Version 2 (EK2) N/A
Goniometry CDE Details
Gross Motor Function Measure (GMFM-88, GMFM-66) N/A
Jebsen-Taylor Hand Function Test N/A
Modified Hammersmith Functional Motor Scale for Children with Spinal Muscular Atrophy (MHFMS-SMA/MHFMS-Extend) N/A
Nine Hole Peg Test N/A
North Star Ambulatory Assessment (NSAA) N/A
Pediatric Evaluation of Disability Inventory (PEDI) N/A
Stair Climb N/A
Timed Up and Go (TUG) N/A
Vignos Lower Extremity Scale N/A
Muscle Strength Testing
CRF Module/Guidance CDEs
Grip Strength Fatigue CDE Details
Hand Held Dynamometry CDE Details
Manual Muscle Testing-Using the Medical Research Council Muscle Grading Scale CDE Details
Maximum Voluntary Isometric Contraction (MVIC) N/A
Pinch Strength N/A
Pulmonary Function Testing/Respiratory Status
CRF Module/Guidance CDEs
Measures of Gas Exchange CDE Details
Pulmonary Assessment Master Outline and Guidelines for Use N/A
Pulmonary Function Testing CDE Details
Performance Measures
CRF Module/Guidance CDEs
Motor Function Measure (MFM) N/A
Quality of Life/Patient Reported Outcomes
CRF Module/Guidance CDEs
Patient-Reported Outcomes Measurement Information System (PROMIS) N/A
Quality of Life in Neurological Disorders (Neuro-QOL) N/A
Neuropsychological Testing
CRF Module/Guidance CDEs
Peabody Picture Vocabulary Test 4th Edition (PPVT-4) N/A
Purdue Pegboard N/A
Wechsler Abbreviated Scale of Intelligence (WASI) N/A
Wechsler Individual Achievement Test-III (WIAT-III) N/A
Wechsler Intelligence Scale for Children-V (WISC-V) N/A
Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III) N/A

Prospective Assessments for Suicidal Ideation and Behavior

Investigators should review the FDA’s "Guidance for Industry: Suicidal Ideation and Behavior: Prospective Assessment of Occurrence in Clinical Trials” for the most up-to-date information about suicidal ideation and behavior. One scale that FDA suggests is the Columbia Suicide Severity Rating Scale (C-SSRS) (available at https://cssrs.columbia.edu/).

Instrument Notice of Copyright Information

The NINDS CDE Team does not post proprietary instruments/scales recommended by the CDE Working Groups on this website. This includes, but is not limited to, copyrighted or trademarked instruments/scales. Information about recommended instruments can be found in the Notice of Copyright (NOC) documents under ‘CRF Module/Guidance’ on each disorder’s data standards page. For any questions regarding these instruments/scales please contact the corresponding owner/author. The NINDS CDE Team is not responsible for the availability or content of these external sites, nor does the NINDS CDE Team endorse, warrant or guarantee the products, services or information described or offered at these other internet sites.

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

Roster

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

CMD Working Group

  • Angela Giacoletti Argento, PhD
    University of Michigan Hospital and Health Systems, Ann Arbor, Michigan
  • Carsten Bonnemann, 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

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

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