This application seeks funding for the Data Management and Coordinating Center (DMCC) for the Rare Diseases Clinical Research Network (RDCRN). The applicant, Dr. Krischer, has served as the Principal Investigator for the DMCC for the last 10 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 two grant cycles to accommodate the more than 24,000 subjects who have been enrolled on 128 studies (86 of which are currently accruing), 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 websit for web-based recruitment which receives over 7 million hits per year at present and a 14,000+ patient contact registry enhanced for subjects seeking information on rare diseases and 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 clinicl 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.
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.
|Killian Jr, John T; Lane, Jane B; Lee, Hye-Seung et al. (2016) Caretaker Quality of Life in Rett Syndrome: Disorder Features and Psychological Predictors. Pediatr Neurol 58:67-74|
|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|
|Tamura, Roy N; Krischer, Jeffrey P; Pagnoux, Christian et al. (2016) A small n sequential multiple assignment randomized trial design for use in rare disease research. Contemp Clin Trials 46:48-51|
|Kermani, Tanaz A; Warrington, Kenneth J; Cuthbertson, David et al. (2015) Disease Relapses among Patients with Giant Cell Arteritis: A Prospective, Longitudinal Cohort Study. J Rheumatol 42:1213-7|
|Fridman, V; Bundy, B; Reilly, M M et al. (2015) CMT subtypes and disease burden in patients enrolled in the Inherited Neuropathies Consortium natural history study: a cross-sectional analysis. J Neurol Neurosurg Psychiatry 86:873-8|
|Johnson, Nicholas E; Heatwole, Chad R; Dilek, Nuran et al. (2014) Quality-of-life in Charcot-Marie-Tooth disease: the patient's perspective. Neuromuscul Disord 24:1018-23|
|Krischer, Jeffrey P; Gopal-Srivastava, Rashmi; Groft, Stephen C et al. (2014) The Rare Diseases Clinical Research Network's organization and approach to observational research and health outcomes research. J Gen Intern Med 29 Suppl 3:S739-44|