This application seeks funding for the Data Management and Coordinating Center (DMCC) (formerly known as Data Technology Coordinating Center, DTCC) for the Rare Diseases Clinical Research Network (RDCRN). The applicant, Dr. Krischer, has served as the Principal Investigator for the DTCC for the last 5 years and seeks to renew the cooperative agreement for the DMCC which supports the Rare Diseases Clinical Research Network (RDCRN). The DMCC propose to extend the systems, processes, and procedures developed successfully over the last grant cycle to accommodate the 3000 subjects enrolled on 32 current studies, contingent upon the successful re-competition of their associated clinical research consortia, addition of new studies reflecting the growth of the network, accommodation of federated databases, work with consortia that have pre-existing infrastructure (registries, patient databases, etc.) and registries, provide a user friendly website for web-based recruitment which receives over 3.4 million hits per year at present and a 4000+ member contact registry enhanced for subjects seeking enrollment on clinical trials. We will continue development of new technologies to support scalability and generalizability and tools for cross-disease data mining. Our international clinical information network is secure providing coordinated data management services for collection, storage and analysis of diverse data types from multiple diseases and geographically disparate locations and a portal for the general public and larger community of clinical investigators.

Public Health Relevance

The proposed DMCC will facilitate clinical research in rare diseases by providing a test-bed for distributed clinical data management that incorporates novel approaches and technologies for data management, data mining, and data sharing across rare diseases, data types, and platforms;and access to information related to rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the lay public.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZRG1-HOP-Y (50))
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Stewart, Randall R
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University of South Florida
Schools of Medicine
United States
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Griffith, Linda M; Cowan, Morton J; Notarangelo, Luigi D et al. (2016) Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol 138:375-85
Merkel, Peter A; Manion, Michele; Gopal-Srivastava, Rashmi et al. (2016) The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network. Orphanet J Rare Dis 11:66
Haddad, Elie; Allakhverdi, Zoulfia; Griffith, Linda M et al. (2014) Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol 133:597-9
Griffith, Linda M; Cowan, Morton J; Notarangelo, Luigi D et al. (2014) Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol 133:335-47
Shearer, William T; Dunn, Elizabeth; Notarangelo, Luigi D et al. (2014) Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol 133:1092-8
Kwan, Antonia; Abraham, Roshini S; Currier, Robert et al. (2014) Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States. JAMA 312:729-38
Ashizawa, Tetsuo; Figueroa, Karla P; Perlman, Susan L et al. (2013) Clinical characteristics of patients with spinocerebellar ataxias 1, 2, 3 and 6 in the US; a prospective observational study. Orphanet J Rare Dis 8:177
Monach, Paul A; Warner, Roscoe L; Tomasson, Gunnar et al. (2013) Serum proteins reflecting inflammation, injury and repair as biomarkers of disease activity in ANCA-associated vasculitis. Ann Rheum Dis 72:1342-50
Monach, Paul A; Kümpers, Philipp; Lukasz, Alexander et al. (2012) Circulating angiopoietin-2 as a biomarker in ANCA-associated vasculitis. PLoS One 7:e30197
Monach, Paul A; Tomasson, Gunnar; Specks, Ulrich et al. (2011) Circulating markers of vascular injury and angiogenesis in antineutrophil cytoplasmic antibody-associated vasculitis. Arthritis Rheum 63:3988-97

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