Facioscapulohumeral 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)

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

Facioscapulohumeral Muscular Dystrophy Highlight Summary: Overview of all Facioscapulohumeral 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
Quality of Life in Neurological Disorders (Neuro-QOL) N/A
National Institutes of Health (NIH) Toolbox N/A
Patient-Reported Outcomes Measurement Information System (PROMIS) N/A
Overview of Working Group Recommendations
Summary Documents
CRF Module/Guidance CDEs
Summary of Recommendations for FSHD N/A
Participant Characteristics
Demographics
CRF Module/Guidance CDEs
General Core CDE Details
Demographics CDE Details
Social Status
CRF Module/Guidance CDEs
Social Status CDE Details
Participant History and Family History
Epidemiology/Environmental History
CRF Module/Guidance CDEs
World Health Organization Disability Assessment Schedule (WHODAS) N/A
General Health History
CRF Module/Guidance CDEs
Family History CDE Details
Medical History CDE Details
Pregnancy CDE Details
Assessments and Examinations
Imaging Diagnostics
CRF Module/Guidance CDEs
Dual X-Ray Absorptiometry (DXA) CDE Details
Magnetic Resonance Imaging (MRI) CDE Details
MRI Quantitative Measurements Guidelines N/A
Physical/Neurological Examination
CRF Module/Guidance CDEs
Electrical Impedance Myography (EIM) N/A
Polysomnography CDE Details
Non-Imaging Diagnostics
CRF Module/Guidance CDEs
Electrocardiogram (ECG) CDE Details
Laboratory Tests and Biospecimens/Biomarkers
CRF Module/Guidance CDEs
Laboratory Tests and Tracking CDE Details
Laboratory Tests Recommendations Table and Guidelines for Use N/A
Mutation Analysis CDE Details
Treatment/Intervention Data
Therapies
CRF Module/Guidance CDEs
Respiratory Interventions CDE Details
Drugs
CRF Module/Guidance CDEs
Prior and Concomitant Medications CDE Details
Outcomes and End Points
Functional Outcomes
CRF Module/Guidance CDEs
2 Minute Walk Test N/A
6 Minute Walk Test N/A
Goniometry CDE Details
Stair Climb N/A
Timed Up and Go (TUG) N/A
Pain
CRF Module/Guidance CDEs
Brief Pain Inventory (BPI) Short Form N/A
McGill Pain Questionnaire (MPQ) N/A
Pain Assessment in FSHD Guidelines for Use N/A
Muscle Strength Testing
CRF Module/Guidance CDEs
Grip Strength 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
Quality of Life/Patient Reported Outcomes
CRF Module/Guidance CDEs
Individualized Neuromuscular Quality of Life Questionnaire (INQoL) N/A
Quality of Life in Neurological Disorders (Neuro-QOL) N/A
Short Form 36-Item Health Survey (SF-36) 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

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 Facioscapulohumeral Muscular Dystrophy (FSHD) CDEs, the FSHD 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.

Roster

The Facioscapulohumeral Muscular Dystrophy (FSHD) CDE Working Group has developed the CDEs for their recommendations. The FSHD 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 FSHD research.

Complete Facioscapulohumeral Muscular Dystrophy (FSHD) CDE Roster

The complete FSHD CDE Working Group roster is shown below.

FSHD Working Group

FSHD Working Group

  • John T. Kissel, MD
    Ohio State University, Columbus, Ohio
  • George W.A.M. Padberg, MD
    Nijmegen, Netherlands
  • Enzo Ricci, MD
    Universita Cattolica del Sacro Cuore, Rome, Italy
  • Sabrina Sacconi, MD
    Centre Hopitalier Universitaire de Nice Sophia Antipolis, Nice, France
  • Jeffrey Statland, MD
    University of Kansas Medical Center, Kansas
  • Rabi Tawil, MD
    University of Rochester Medical Center, Rochester, New York
  • Baziel G. M. van Engelen, MD
    Neuromuscular Centre Nijmegen, Nijmegen, Netherlands
  • Kathryn R. Wagner, MD, PhD
    Kennedy Krieger Institute, Baltimore, Maryland

NIH Coordinating Team

NIH Coordinating Team

  • Robin Conwit, MD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, MD, USA
  • 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, MD, USA
  • Yaffa Rubenstein, PhD
    National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Bethesda, Maryland
  • Tiina 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
    The Emmes Corporation
  • Robin Feldman, BS, MBA
    The Emmes Corporation
  • Muniza Sheikh, MS, MBA
    The Emmes Corporation

Publications

A publication related to the Facioscapulohumeral Muscular Dystrophy CDE initiative will be written at a later date. 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.

Facioscapulohumeral CDE Revision History Document