Common data elements for clinical research in Friedreich's ataxia |
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| Authors: | David R. Lynch MD PhD Massimo Pandolfo MD Jorg B. Schulz MD Susan Perlman MD Martin B. Delatycki MD PhD R. Mark Payne MD Robert Shaddy MD Kenneth H. Fischbeck MD Jennifer Farmer MS Paul Kantor MD Subha V. Raman MD Lisa Hunegs MSW MPH Joanne Odenkirchen MPH Kristy Miller MPH Petra Kaufmann MD MSC |
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| Affiliation: | 1. Departments of Neurology and Pediatrics, University of Pennsylvania and the Children's Hospital of Philadelphia, , Philadelphia, Pennsylvania;2. Université Libre de Bruxelles, , Brussells, Belgium;3. Department of Neurology and JARA Brain, University Hospital, , RWTH Aachen, Germany;4. University of California, , Los Angeles, California, USA;5. Murdoch Children's Research Institute, , Melbourne, Australia;6. Indiana University, , Indianapolis, Indiana, USA;7. Department of Pediatrics, Division of Cardiology, University of Pennsylvania, the Children's Hospital of Philadelphia, , Philadelphia, Pennsylvania, USA;8. National Institute of Neurological Disorders and Stroke, National Institutes of Health, , Bethesda, Maryland, USA;9. Friedreich's Ataxia Research Alliance, , Downingtown, Pennsylvania, USA;10. Hospital for Sick Children, , Toronto, Ontario, Canada;11. The Ohio State University, , Columbus, Ohio, USA;12. KAI Research, Inc., , Rockville, Maryland, USA |
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| Abstract: | To reduce study start‐up time, increase data sharing, and assist investigators conducting clinical studies, the National Institute of Neurological Disorders and Stroke embarked on an initiative to create common data elements for neuroscience clinical research. The Common Data Element Team developed general common data elements, which are commonly collected in clinical studies regardless of therapeutic area, such as demographics. In the present project, we applied such approaches to data collection in Friedreich's ataxia (FRDA), a neurological disorder that involves multiple organ systems. To develop FRDA common data elements, FRDA experts formed a working group and subgroups to define elements in the following: ataxia and performance measures; biomarkers; cardiac and other clinical outcomes; and demographics, laboratory tests, and medical history. The basic development process included identification of international experts in FRDA clinical research, meeting by teleconference to develop a draft of standardized common data elements recommendations, vetting of recommendations across the subgroups, and dissemination of recommendations to the research community for public comment. The full recommendations were published online in September 2011 at http://www.commondataelements.ninds.nih.gov/FA.aspx . The subgroups′ recommendations are classified as core, supplemental, or exploratory. Template case report forms were created for many of the core tests. The present set of data elements should ideally lead to decreased initiation time for clinical research studies and greater ability to compare and analyze data across studies. Their incorporation into new, ongoing studies will be assessed in an ongoing fashion to define their utility in FRDA. © 2012 Movement Disorder Society |
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| Keywords: | cerebellum dorsal root ganglion mitochondrion clinical measure NINDS Common Data Elements |
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