Abstract Details

Title Updates to the National Institute of Neurological Disorders and Stroke (NINDS) Friedreich’s Ataxia (FA) Common Data Element (CDE) Recommendations: 2018 FA Working Group Review

Topic Movement Disorders

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 10-007

Objective Through the development of Friedreich’s Ataxia (FA)-specific common data elements (CDEs), the National Institute of Neurological Disorders and Stroke (NINDS) CDE project strives to improve data collection by increasing efficiency to reduce study start-up time, improving data quality, and facilitating data sharing/meta-analyses. The CDEs are dynamic and are updated as needed with new scientific findings.

Background The FA CDEs were developed in 2011 by an international group of subject matter experts. Recommendations were stratified by classifications: Core (required), Supplemental-Highly Recommended (strongly encouraged based on study type), Supplemental (commonly collected, but not required), and Exploratory (reasonable to fill in gaps, but require further validation). An FA Oversight Committee (OC) was subsequently formed to review user feedback and updates to the CDEs based on research advancements. The OC convened in January 2018 and determined that a comprehensive review was needed to update the FA CDEs.

Design/Methods Domain-specific subgroups were convened to review the current FA CDEs: Ataxia and Performance Measures; Biomarkers; Cardiac and Clinical Outcomes; and Imaging. Subgroup members focused on ensuring the accuracy of current classifications, providing any new elements/measures to be included, and determining a context for use for Supplemental – Highly Recommended CDEs.

Results Updates to the FA CDEs will include classification revisions and a new Brain MRI case report form. The FA OC and subgroups will review the draft recommendations prior to having a public review on the NINDS CDE website. Updates to the FA CDEs will be posted once comments and reviews have been incorporated in 2019.

Conclusions NINDS encourages the use of CDEs for all clinical research in neuroscience. To ensure that the FA CDEs are a dynamic and useful resource, the recommendations are updated periodically based on the current state of science and user input.

Authors/Disclosures
Katelyn E. Gay (The Emmes Company, LLC) PRESENTER	Ms. Gay has received personal compensation for serving as an employee of The Emmes Company, LLC.
	No disclosure on file
Muniza Sheikh (The Emmes Company, LLC)	Ms. Sheikh has received personal compensation for serving as an employee of The Emmes Company.
Joy R. Esterlitz (The Emmes Corp)	No disclosure on file
David R. Lynch, MD, PhD (Children's Hospital of Philadelphia)	The institution of Dr. Lynch has received research support from reata. The institution of Dr. Lynch has received research support from PTC. Dr. Lynch has received intellectual property interests from a discovery or technology relating to health care.
Massimo Pandolfo, MD, FAAN	Dr. Pandolfo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Design Therapeutics . Dr. Pandolfo has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Pandolfo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Bio. Dr. Pandolfo has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurocrine. Dr. Pandolfo has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Design Therapeutics. Dr. Pandolfo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Larimar. Dr. Pandolfo has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology Genetics. Dr. Pandolfo has stock in Voyager Therapeutics. The institution of Dr. Pandolfo has received research support from Freidreich Ataxaia Research Alliance. Dr. Pandolfo has a non-compensated relationship as a Scientific Advisory Board with Friedreich Ataxia Research Alliance that is relevant to AAN interests or activities. Dr. Pandolfo has a non-compensated relationship as a Member-Meeting Management Committee with AAN that is relevant to AAN interests or activities.
	No disclosure on file
Codrin I. Lungu, MD, FAAN	Dr. Lungu has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier, inc..
	No disclosure on file

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