The Columbia PCC for CureGN: the Cure Glomerulonephropathy network

哥伦比亚 PCC for CureGN：治愈肾小球肾病网络

• 批准号: 10212101
• 负责人: ALI G GHARAVI
• 金额: $ 16.2万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-16 至 2022-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10212101
• 关键词: Achievement; Address; Adult; Affect; Ancillary Study; Award; Basic Science; Biocompatible Materials; Biological Products; Blood; Caring; Child; Childhood; Classification; Clinical; Clinical Data; Clinical Research; Collaborations; Collection; Communication; Communities; DNA; Data; Data Analyses; Data Coordinating Center; Data Set; Databases; Development; Diagnosis; Disease; End stage renal failure; Enrollment; Ethnic Origin; Etiology; Focal Segmental Glomerulosclerosis; Fostering; Foundations; Funding Agency; Gap Junctions; Goals; Grant; Health; IGA Glomerulonephritis; Immunoglobulin A; Individual; Industry; Infrastructure; International; Investigation; Joints; Kidney; Kidney Diseases; Knowledge; Longitudinal observational study; Manuscripts; Membranous Glomerulonephritis; Michigan; Midwestern United States; Modernization; Morbidity - disease rate; Natural History; North Carolina; Participant; Pathogenesis; Pathologic; Pathway interactions; Patient Outcomes Assessments; Patient Recruitments; Patients; Pattern; Pennsylvania; Pilot Projects; Policies; Population Heterogeneity; Privatization; Procedures; Prospective cohort study; Protocols documentation; Publications; RNA; Race; Renal glomerular disease; Research; Research Personnel; Resources; Scientist; Site; Standardization; Structure; Time; Training; Translational Research; United States National Institutes of Health; Universities; Urine; Work; burden of illness; career development; clinical center; cohort; collaborative environment; data sharing; design; digital pathology; disorder subtype; experience; follow-up; improved; innovation; instrument; kidney biopsy; meetings; mortality; multidisciplinary; next generation; novel; outcome forecast; patient advocacy group; personalized care; precision medicine; predict clinical outcome; predicting response; programs; prospective; public-private partnership; recruit; repository; research and development; response; retention rate; whole genome; working group

Project Summary/Abstract Primary glomerular diseases including minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), immunoglobulin A nephropathy (IGAN), and membranous nephropathy (MN) account for 10% of end-stage renal disease and are associated with significant morbidity and mortality in both adults and children. Despite the significant disease burden associated with these heterogeneous disorders, disease pathogenesis, natural history, predictors of clinical outcomes and predictors of response to therapy remain incompletely defined. To address these gaps in knowledge, the CureGN consortium assembled a large consortium of 70 clinical study sites, nephrologists, scientists with diverse expertise, affected patients and advocacy groups, the biopharmaceutical industry, federal funding agencies, and regulatory agencies. CureGN is conducting a prospective, longitudinal observational study, successfully enrolling and retaining a race and ethnicity-balanced cohort of nearly 2400 adult and pediatric participants with IgAN, FSGS, MN and MCD. We propose to maintain and enhance the CureGN Consortium infrastructure and its ancillary study program to accelerate patient-relevant glomerular disease research. We will continue our core prospective, longitudinal observational study of glomerular disease patients, enrolling additional subjects by a recruit-to-replace strategy to maintain an active cohort of 2400, while banking and curating high quality clinical data and biomaterials. This foundational work is being conducted by a refined study administrative structure and a well-functioning collaboration between the Data Coordinating Center (at Arbor Research Collaborative for Health and the University of Michigan) and four Participating Clinical Centers (managed at the University of Pennsylvania, Columbia University, University of North Carolina, and the Midwest Pediatric Consortium). CureGN sits at the nexus of multiple domains that are collaborating in overcoming the barriers to improving care for glomerular disease patients. By its now experienced scientific working groups, its publications committee, and its ancillary study infrastructure, by enabling modern analytical approaches in multiple relatable knowledge domains, by administering a research opportunity pool in conjunction with NephCure Kidney International, by continuing refinement of study instruments, by enhancing career development activities, and by convening patient and public-private collaborations for validating and implementing precision medicine-based therapies, the resource created by CureGN should accelerate improving care of glomerular disease patients.

项目摘要/摘要 原发性肾小球疾病，包括最小变化疾病（MCD），局灶性节段性 肾小球硬化（FSG），免疫球蛋白A肾病（Igan）和膜状 肾病（MN）占末期肾脏疾病的10％，与显着有关 成人和儿童的发病率和死亡率。尽管疾病负担很大 与这些异质性疾病，疾病发病机理，自然史，预测有关 临床结果和对治疗反应的预测因素仍未完全定义。到 在知识中解决这些差距，治疗财团组装了一个70的大财团 临床研究地点，肾脏科医生，具有多样专业知识的科学家，患者和 倡导团体，生物制药行业，联邦资助机构和监管 机构。 Curegn正在进行一项前瞻性，纵向观察性研究，成功地 招收和保留近2400名成人和儿科的种族和种族平衡队列 IGAN，FSGS，MN和MCD的参与者。我们建议维护和增强治疗 财团基础设施及其辅助研究计划，以加速患者与患者相关 肾小球疾病研究。我们将继续我们的核心前瞻性，纵向观察 肾小球疾病患者的研究，通过招募策略招募其他受试者 维持2400的主动队列，同时融合和策划高质量的临床数据和 生物材料。这项基础工作是由精致的研究行政结构进行的 以及数据协调中心（Arbor Research）之间的功能合理的合作 卫生和密歇根大学的合作）和四个参与的临床中心 （北卡罗来纳大学宾夕法尼亚大学，哥伦比亚大学的宾夕法尼亚大学管理 和中西部小儿财团）。治疗位于多个领域的联系 协作克服改善肾小球疾病患者护理的障碍。通过它的 现在经验丰富的科学工作组，其出版委员会及其辅助研究 基础架构，通过在多种相关知识中启用现代分析方法 域，通过与Nephcure肾脏一起管理研究机会池 国际，通过继续改进学习工具，通过增强职业发展 活动，并通过召集患者和公私合作进行验证和 实施基于精确医学的疗法，Curegn创建的资源应 加速改善肾小球疾病患者的护理。