CureGN-Penn PCC

CureGN-宾夕法尼亚PCC

• 批准号: 10656280
• 负责人: LAWRENCE B. HOLZMAN
• 金额: $ 104.68万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 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 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10656280
• 关键词: Acceleration; Achievement; Address; Adult; Advocacy; Affect; Ancillary Study; Award; Basic Science; Biocompatible Materials; 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 and Segmental Glomerulosclerosis; Fostering; Funding Agency; 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; Prediction of Response to Therapy; Privatization; Procedures; Prognosis; 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; biopharmaceutical industry; 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; multi-ethnic; multidisciplinary; next generation; novel; patient engagement; personalized care; precision medicine; predict clinical outcome; programs; prospective; public-private partnership; recruit; repository; retention rate; treatment response; whole genome; working group

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.

原发性肾小球疾病，包括微小病变（MCD）、局灶性节段性肾小球疾病 肾小球硬化症（FSGS）、免疫球蛋白A肾病（IGAN）和膜性肾病 肾病（MN）占终末期肾病的10%，并且与显著的 成人和儿童的发病率和死亡率尽管疾病负担很重 与这些异质性疾病、疾病发病机制、自然史、预测因素相关 的临床结果和治疗反应的预测因素仍然不完全确定。到 为了解决这些知识上的差距，CureGN财团组建了一个由70名成员组成的大型财团， 临床研究中心、肾病学家、具有不同专业知识的科学家、受影响的患者和 倡导团体、生物制药行业、联邦资助机构和监管机构 社. CureGN正在进行一项前瞻性、纵向观察性研究， 招募并保留了一个由近2400名成人和儿童组成的种族和民族平衡的队列， 参与者与IgAN，FSGS，MN和MCD。我们建议维持和加强CureGN 联盟基础设施及其辅助研究计划，以加速患者相关 肾小球疾病研究。我们将继续我们的核心前瞻性，纵向观察 肾小球疾病患者研究，通过招募替代策略招募额外受试者 保持2400名活跃的队列，同时储存和管理高质量的临床数据， 生物材料这项基础性工作正在由一个完善的研究行政结构进行 以及数据协调中心（Arbor Research）与 协作健康和密歇根大学）和四个参与临床中心 （管理于宾夕法尼亚大学、哥伦比亚大学、北卡罗来纳州大学、 和中西部儿科联盟）。CureGN位于多个域的连接处， 合作克服改善肾小球疾病患者护理的障碍。由其 现在经验丰富的科学工作组、其出版物委员会及其辅助研究 基础设施，通过在多种相关知识中启用现代分析方法 通过与NephCure Kidney联合管理研究机会库， 国际化，通过不断完善学习工具，通过加强职业发展 活动，并召集病人和公私合作， 实施基于精确医学的治疗，CureGN创建的资源应该 加快改善肾小球疾病患者的护理。