Data Coordinating Center for the Halt-Polycystic Kidney Disease Trials

描述（由申请人提供）：halt -多囊肾病（PKD）试验包括2个完全入组的随机对照试验（A和B），在7个临床站点进行，由一个中心成像设施、一个药物分配中心和2个中心实验室支持。HALT-PKD研究A采用2x2因子设计评估肾素-血管紧张素-醛固酮系统（RAAS）阻断和2个水平的血压控制对558例高正常或高血压PKD患者疾病结构性进展的影响，估计肾小球滤过率（GFR）为60 ml/min/1.73m2。主要终点是在0、24和48个月时测量的总肾容量（TKV）。HALT- PKD研究B评估了486例GFR为30-60 ml/min/1.73m2的高血压PKD患者RAAS阻断对疾病进展的影响。主要终点是由eGFR、ESRD或死亡减少50%定义的联合终点。参与者被跟踪了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的短随访时间（48个月）可能不足以看到肾功能的变化。在研究B中，观察到的5年终点数低于预期，为结果降低25%提供了动力。由于这些新发现和中期分析，DSMB批准将这两项研究延长至2014年7月，以允许研究a（60个月）的额外措施和研究b的5-8年随访。我们建议继续作为HALT-PKD DCC， 1)与研究研究者合作，管理方案和法规遵从性，促进数据、图像和生物标本的传输，并支持HALT-PKD活动进行质量控制、终点裁决；血压管理；2)维护基于web的数据管理系统，包括数据跟踪、录入、质量控制和报告生成；3)进行中期和最终统计分析，以支持研究目标，包括研究A和研究b的未来初步分析

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