Organized into domains often used in clinical studies, data standards
include:
- CDEs
- CRF Modules logically organize CDEs for data collection
- Guidelines to provide further information about the CDEs.
- Recommended Instruments spreadsheets with details
(descriptions, scoring, references, etc.) for all recommended proprietary instruments/
scales/ tests. If proprietary instruments/scales/tests are made available for use,
they are populated in the table below.
For your reference, a zip file containing all of the Friedreich’s Ataxia (FA) CDE
CRF modules can be downloaded below.
Download FA CDE Recommendations
The outline that follows includes all the CDEs associated with the CRF modules,
organized by domain and sub-domain.
Participant/Subject Characteristics
Participant/Subject Characteristics
| CRF Module/Guideline by Sub-Domain |
Download |
CDEs |
© or TM |
| Demographics |
| Demographics
|
45KB DOC |
HTML |
|
| Social Status |
| Social Status
|
57KB DOC |
HTML |
|
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Participant/Subject History and Family History
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Disease/Injury Related Events
Disease/Injury Related Events
| CRF Module/Guideline by Sub-Domain |
Download |
CDEs |
© or TM |
| History of Disease/Injury Event |
| Medical History of Friedreich's Ataxia
|
73KB DOC |
HTML |
|
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Assessments and Examinations
Assessments and Examinations
| CRF Module/Guideline by Sub-Domain |
Download |
CDEs |
© or TM |
| Physical/Neurological Examination |
| Physical Examination
|
77KB DOC |
HTML |
|
| Vital Signs and Other Body Measures |
| Vital Signs
|
64KB DOC |
HTML |
|
| Laboratory Tests and Biospecimens/Biomarkers |
| Laboratory Tests
|
291KB DOC |
HTML |
|
| Plasma Collection Guidelines
|
50KB DOC |
N/A |
|
| Urine Collection Guidelines
|
49KB DOC |
N/A |
|
| Imaging Diagnostics |
| Cardiac and Magnetic Resonance Imaging
|
43KB DOC |
HTML |
|
| Non-Imaging Diagnostics |
| Electrocardiogram
|
61KB DOC |
HTML |
|
| Echocardiogram
|
56KB DOC |
HTML |
|
| Holter Exam
|
45KB DOC |
HTML |
|
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Treatment/Intervention Data
Note: The General CDE Standards contain additional useful CRF Modules and CDEs for this domain.
Treatment/Intervention Data
| CRF Module/Guideline by Sub-Domain |
Download |
CDEs |
© or TM |
| Drugs |
| Prior and Concomitant Medications
|
90KB DOC |
HTML |
|
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Protocol Experience
Note: The General CDE Standards contain useful CRF Modules and CDEs for this domain.
Safety Data
Note: The General CDE Standards contain useful CRF Modules and CDEs for this domain.
Outcomes and End Points
The CDEs from copyright-protected measures are intentionally excluded from this site unless permission was received from the copyright holder. If the measure is not included, please contact the publisher or person who holds the copyright to obtain a copy of the measure.
Outcomes and End Points
| CRF Module/Guideline by Sub-Domain |
Download |
CDEs |
© or TM |
| Summary of All Outcome Recommendations |
| FA CDE Instrument Recommendations by Outcome Domain and Classification
|
XLS  |
N/A |
|
| Activities of Daily Living/Performance |
| Activities of Daily Living and Gait
|
113KB DOC |
HTML |
|
| Ataxia and Performance Measures |
| 9-Hole Peg Test
|
185KB PDF |
HTML |
|
| Bladder Control Scale (BLCS)
|
17KB PDF |
HTML |
|
| Bowel Control Scale (BWCS)
|
NOC |
HTML |
|
| Friedreich's Ataxia Impact Scale (FAIS)
|
1062KB PDF |
HTML |
 |
| Friedreich's Ataxia Rating Scale (FARS)
|
68KB PDF |
HTML |
|
| Impact of Visual Impairment Scale (IVIS)
|
17KB PDF |
HTML |
|
| Low Contrast Letter Acuity
|
117KB DOC |
HTML |
|
| Modified Fatigue Impact Scale (MFIS)
|
18KB PDF |
HTML |
|
| MOS Pain Effects Scale (PES)
|
17KB PDF |
HTML |
|
| Scale for the Assessment and Rating of Ataxia (SARA)
|
1023KB PDF |
HTML |
|
| Tardieu Scale
|
66KB DOC |
HTML |
|
| Timed 25-Foot Walk (T25FW)
|
185KB PDF |
HTML |
|
| Quality of Life |
| 36-Item Short Form Health Survey (SF-36)
|
45KB DOC |
HTML |
|
| Clinical Event End Points |
| Cardiac End Points
|
44KB DOC |
HTML |
|
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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
http://www.cssrs.columbia.edu, see additional C-SSRS information for
background materials provided to the NINDS CDE project by Dr. Posner) or another
instrument that maps to the Columbia Classification Algorithm for Suicide Assessment
(C-CASA) (Posner K, Oquendo MA, Gould M, Stanley B, Davies M. Am J Psychiatry. 2007 Columbia Classification Algorithm of
Suicide Assessment (C-CASA): classification of suicidal events in the FDA's pediatric suicidal risk analysis of antidepressants. Jul;164(7):1035-43. PMID: 17606655).
Accessibility Statement
The NINDS CDE Team has requested, and in many cases received, permission to post
proprietary instruments/scales recommended by the CDE Working Groups to this site.
When permission is granted, the documents are posted in the exact format they were
received in order to maintain the validity of the instrument. If approval is not
granted for the instrument to be reproduced on this site, with the author/copyright
holder’s permission, a link is posted to an external Web site where users can get
more information about the instrument. If you have difficulty accessing either the
proprietary instruments/scales or the external links, please contact the
NINDS CDE Project Officer, Joanne Odenkirchen.
Download readers:
To develop the Friedreich’s Ataxia (FA) CDEs, the
FA CDE Working Group divided into subgroups to focus on identifying and
defining data elements in the domains of:
- Ataxia and Performance Measures
- Biomarkers
- Cardiac and Clinical Outcomes
- Demographics, Medical History/ Prior Health Status, Laboratory Tests/ Vital Signs
Data standards include CDEs as well as case report form (CRF) Modules and Guidelines.
The CRF Modules logically organize the CDEs for data collection, while the Guidelines
provide further information about the CDEs. The CDEs, CRF Modules, and Guidelines
presented are organized into domains which are intuitive and common in clinical
research studies.
The Friedreich’s Ataxia CDE Working Group is currently comprised of four different
subgroups. Chairs were appointed to lead the overall Working Group and the individual
subgroups. The Working Group members actively develop the CDEs for their specific
subgroups and also have an opportunity to review and comment on the recommendations
of the other subgroups. The FA CDE Working Group is supported by the
NINDS CDE Team. The complete FA CDE Working Group roster and the rosters
by Subgroup are shown below.
Complete Friedreich's Ataxia CDE Working
Group Roster 
The Working Group members serve on either one or two subgroups. The subgroups began
meeting by teleconference in August 2010 and continued to hold calls every four
to six weeks through early 2011 to define the CDEs for their domains and to recommend
standardized, validated instruments for Friedreich’s Ataxia research.
Working Group Co-Chairs
- David Lynch, MD, PhD - University of Pennsylvania
- Massimo Pandolfo, MD - Université Libre de Bruxelles
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Ataxia and Performance Measures
- Martin Delatycki, MD, PhD - Murdoch Institute, Royal Children's Hospital
- David Lynch, MD, PhD - University of Pennsylvania
- Susan Perlman, MD - University of California Los Angeles
- Jorg Schulz, MD - University Medical Center, RWTH Aachen
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Biomarkers
- Kurt Fischbeck, MD - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Massimo Pandolfo, MD - Université Libre de Bruxelles
- Mark Payne, MD - Indiana University
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Cardiac and Clinical Outcomes
- Jennifer Farmer, MS - Friedreich's Ataxia Research Alliance
- Paul Kantor, MD - Hospital for Sick Children
- Mark Payne, MD - Indiana University
- Subha Raman, MD - The Ohio State University Medical Center
- Robert Shaddy, MD - Children's Hospital of Philadelphia
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Demographics, Medical History/ Prior Health Status, Laboratory Tests/ Vital Signs
- Jennifer Farmer, MS - Friedreich's Ataxia Research Alliance
- David Lynch, MD, PhD - University of Pennsylvania
- Jorg Schulz, MD - University Medical Center, RWTH Aachen
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NINDS CDE Team
- Wendy Galpern, MD, PhD - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Petra Kaufmann, MD, MSc - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Joanne Odenkirchen, MPH - National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
- Sherita Ala'i, MS - The EMMES Corporation, Rockville, Maryland, USA (2013 - present)
- Matthew Beyers, MS - The EMMES Corporation, Rockville, Maryland, USA (2013 - present)
- Pamela West - The EMMES Corporation, Rockville, Maryland, USA (2013 - present)
- Lisa Hunegs, MSW, MPH - KAI Research, Inc. (An Altarum Company), Rockville, Maryland, USA (2007 - 2012)
- Kristy Miller, MPH - KAI Research, Inc. (An Altarum Company), Rockville, Maryland, USA (2007 - 2012)
- Megan Schmidt - KAI Research, Inc. (An Altarum Company), Rockville, Maryland, USA (2007 - 2012)
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Note: Institutions for all individuals acknowledged in this section
were those they belonged to when they joined the Committee. All Committee
members were asked to voluntarily disclose all potential sources of bias in their
involvement with the NINDS CDE Project. The summary disclosure information is available
upon request by contacting NINDSCDE@emmes.com.
The Friedreich’s Ataxia (FA) CDEs were most recently revised in August 2012. The
document below describes all the revisions incorporated in the August 2012 version
compared to the previous version dated September 2011. Please contact NINDSCDE@emmes.com
if you require further information or have any questions about the revision history.
FA CDE Revision History.xls