Albumin in Acute Stroke (ALIAS) Trial Statistical and Data Coordinating Center

描述（由申请人提供）：正在进行的急性卒中白蛋白（ALIAS）第2部分试验是一项多中心、随机、双盲、安慰剂对照的III期试验，旨在确定2g /kg剂量的25%白蛋白（ALB）治疗急性缺血性卒中（AIS）患者的有效性，这些患者的基线NIHSS评分为6，并且可以在卒中发作后5小时内使用研究药物治疗。具体而言，我们希望确定与安慰剂（生理盐水）治疗相比，ALB治疗是否增加了AIS患者预后良好的比例。在随机分组后3个月测量的NIH卒中量表（NIHSS）得分为0或1，或修改的Rankin量表（mRS）得分为0或1，或两者均为有利结果。该试验目前得到了两项NINDS合作协议的支持，分别是迈阿密大学临床协调中心（PI: Myron D. Ginsberg， MD）和南卡罗来纳医科大学统计和数据协调中心（SDCC） （PI: Yuko Y. Palesch，博士）。ALIAS第二部分试验是在第一部分试验之后进行的，第一部分试验招募了434名受试者。这是由于对第1部分协议进行了一些重大修改，以更好地确保受试者的安全。迄今为止，第2部分试验已招募了1100名受试者中的357名，以大约90%的能力检测ALB治疗组相对于生理盐水治疗组的有利结局比例增加10%。目前，北美约有70个临床站点参与了该试验。SDCC的这一竞争性续期申请的目的是使ALIAS第2部分试验成功完成。（CCC的竞争性续期申请于2010年7月提交，目前正在审查中。）SDCC的目标是继续与临床研究人员合作，通过提供可靠和高效的数据和项目管理来促进试验的实施，并将我们的统计专业知识应用于试验数据的分析。此外，在下一个拨款期内，我们亦计划集中发展新的统计方法及/或修改现有方法，以解决试验所产生的统计问题。

DESCRIPTION (provided by applicant): The ongoing Albumin in Acute Stroke (ALIAS) Part 2 Trial is a multicenter, randomized, double- blinded, placebo-controlled Phase III trial to ascertain the effectiveness of 2 g/kg dose of 25% albumin (ALB) therapy in patients with acute ischemic stroke (AIS) whose baseline NIHSS score is 6 and who can be treated with study drug within 5 hours of stroke onset. Specifically, we wish to determine whether ALB therapy increases the proportion of AIS patients with favorable outcome compared to placebo (saline) therapy. Favorable outcome is defined as either an NIH Stroke Scale (NIHSS) score of 0 or 1, or a modified Rankin Scale (mRS) score of 0 or 1, or both, measured at 3 months from randomization. The Trial currently is supported by two NINDS cooperative agreement grants to the Clinical Coordinating Center at the University of Miami (PI: Myron D. Ginsberg, MD) and the Statistical and Data Coordination Center (SDCC) at the Medical University of South Carolina (PI: Yuko Y. Palesch, PhD). The ALIAS Part 2 Trial follows the Part 1 Trial that recruited 434 subjects. It arose as a result of some major modifications to the Part 1 protocol to better ensure subject safety. To date, the Part 2 Trial has enrolled 357 out of 1,100 subjects needed to detect with approximately 90% power a 10% increase in the proportion of favorable outcome in the ALB therapy group relative to saline therapy group. Approximately 70 clinical sites throughout North America are currently participating in the Trial. The purpose of this competitive renewal application from the SDCC is to bring the ALIAS Part 2 Trial to successful completion. (The competitive renewal application from the CCC was submitted in July 2010 and is under review at this writing.) The aims of the SDCC are to continue to collaborate with the clinical investigators to facilitate the implementation of the Trial by providing reliable and efficient data and project management, and to apply our statistical expertise to the analysis of the Trial data. Furthermore, in the next grant period, we also plan to focus on the development of new statistical methods and/or modification of existing methods to address the statistical issues arising from the Trial. PUBLIC HEALTH RELEVANCE: Stroke is the third leading cause of death and the major cause of chronic disability. In spite of large experimental literature that shows the effectiveness of acute intervention to achieve substantial neuroprotection of ischemically threatened brain, to date, no clinically proven agents exist. Therefore, the aim of the ALIAS Part 2 Trial in identifying ALB as a potential neuroprotective agent is still relevant, and a positive result from the Trial would have a significant impact on the clinical practice of AIS treatment.