Data Coordinating Center for the Halt-Polycystic Kidney Disease Trials

停止多囊肾病试验数据协调中心

• 批准号: 8518014
• 负责人: Charity G Patterson (Moore)
• 金额: $ 125万
• 依托单位: UNIVERSITY OF PITTSBURGH AT PITTSBURGH
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-09-15 至 2015-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8518014
• 关键词: Affect; Aldosterone; American; Angiotensins; Area; Attenuated; Autosomal Dominant Polycystic Kidney; Blood Pressure; Cessation of life; Chymosin; Clinical; Clinical Data; Clinical Trials Data Monitoring Committees; Combined Modality Therapy; Computer Retrieval of Information on Scientific Projects Database; Contractor; Data; Data Analyses; Data Coordinating Center; Databases; Disease Progression; Doctor of Philosophy; Drug Packaging; End stage renal failure; Enrollment; Event; Feedback; Future; Generations; Genetic; Glomerular Filtration Rate; Health; Healthcare; Hypertension; Hypotension; Image; Image Analysis; Individual; Information Systems; Inherited; Intervention; Kidney; Kidney Diseases; Kidney Failure; Laboratories; Manuscripts; Measures; Monitor; Morbidity - disease rate; National Institute of Diabetes and Digestive and Kidney Diseases; Online Systems; Outcome; Participant; Patients; Pharmacologic Substance; Polycystic Kidney Diseases; Protocols documentation; Quality Control; Randomized Controlled Trials; Renal function; Renin-Angiotensin-Aldosterone System; Reporting; Research; Research Design; Research Personnel; Site; Site Visit; Specimen; Structure; System; Testing; Time; Universities; Work; adjudication; biobank; blood pressure regulation; clinical research site; cohort; cost effective; data management; design; drug distribution; efficacy testing; follow-up; meetings; patient safety; primary outcome; repository; satisfaction; statistical center

DESCRIPTION (provided by applicant): The HALT-Polycystic Kidney Disease (PKD) trials comprise 2 fully enrolled randomized controlled trials (A & B) conducted at 7 clinical sites supported by a central imaging facility, a drug distribution center, and 2 central laboratories. HALT-PKD Study A uses a 2x2 factorial design to evaluate the impact of rennin-angiotensin-aldosterone system (RAAS) blockade and 2 levels of blood pressure control on structural progression of disease in 558 high-normal or hypertensive PKD patients with estimated glomerular filtration rate (GFR) >60 ml/min/1.73m2. The primary outcome is total kidney volume (TKV) measured at 0, 24, and 48 months. HALT- PKD Study B evaluates the impact of RAAS blockade on progression of disease in 486 hypertensive PKD patients with estimated GFR 30-60 ml/min/1.73m2. The primary outcome is a combined endpoint defined by >50 percent reduction in eGFR, ESRD, or death. Participants are followed for 4-7 years. For Study A, there is strong evidence to show the impact of TKV on kidney function (GFR) takes several years to manifest implying the short period of follow-up for Study A (48 months) may be insufficient to see changes on kidney function. For Study B, the observed number of endpoints at 5 years is lower than had been predicted to provide power for 25 percent reduction in outcome. As a result of these new findings and interim analyses, the DSMB approved extension of both studies through July 2014 to allow an additional measure for Study A (60 months) and 5-8 years follow-up for study B. We propose to continue to serve as the HALT-PKD DCC by 1) collaborating with study investigators, managing protocol and regulatory compliance, facilitating the transfer of data, images, and bio-specimens, and supporting HALT- PKD activities for quality control, endpoint adjudication, and blood pressure management; 2) maintaining the Web-based data management system that incorporates data tracking, entry, quality control, and report generation; 3) conducting interim and final statistical analyses to support the study aims including the future primary analyses for Study A and Study B. Public

描述（由申请人提供）：HALT-多囊肾病（PKD）试验包括2项完全入组的随机对照试验（A和B），在7个临床中心进行，由一个中心成像机构、一个药物配送中心和2个中心实验室提供支持。HALT-PKD研究A采用2x2析因设计，在558例估计肾小球滤过率（GFR）>60 ml/min/1.73m2的正常高值或高血压PKD患者中评价了肾素-血管紧张素-醛固酮系统（RAAS）阻断和2个水平的血压控制对疾病结构进展的影响。主要结局是在0、24和48个月时测量的肾脏总体积（TKV）。HALT- PKD研究B在486例估计GFR 30-60 ml/min/1.73 m2的高血压PKD患者中评价了RAAS阻断对疾病进展的影响。主要结局是一个联合终点，定义为eGFR、ESRD或死亡降低> 50%。参与者被跟踪4-7年。对于研究A，有强有力的证据表明TKV对肾功能（GFR）的影响需要数年才能显现，这意味着研究A的短期随访（48个月）可能不足以观察到肾功能的变化。对于研究B，5年时观察到的终点数量低于预期，为结局减少25%提供了把握度。由于这些新发现和中期分析，DSMB批准将两项研究延长至2014年7月，以允许研究A（60个月）的额外测量和研究B的5-8年随访。我们建议通过以下方式继续担任HALT-PKD DCC：1）与研究者合作，管理方案和监管合规性，促进数据、图像和生物标本的传输，并支持HALT-PKD的质量控制、终点裁定和血压管理活动; 2）维护基于网络的数据管理系统，该系统包括数据跟踪、输入、质量控制和报告生成; 3）进行中期和最终统计分析以支持研究目的，包括研究A和研究B的未来主要分析。公共