Rationale and design of a multicenter study of selegiline and alpha-tocopherol in the treatment of Alzheimer disease using novel clinical outcomes. Alzheimer's Disease Cooperative Study.
Rationale and design of a multicenter study of selegiline and alpha-tocopherol in the treatment of Alzheimer disease using novel clinical outcomes. Alzheimer's Disease Cooperative Study.
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司来吉兰和α-生育酚治疗阿尔茨海默病的多中心研究的基本原理和设计，采用新颖的临床结果。
DOI：
10.1097/00002093-199601030-00004
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发表时间：
1996
期刊：
影响因子：
--
通讯作者：
Thal,LJ
中科院分区：
文献类型：
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作者：
Sano,M;Ernesto,C;Klauber,MR;Schafer,K;Woodbury,P;Thomas,R;Grundman,M;Growdon,J;Thal,LJ
本报告描述了使用司来吉兰（10 mg/天）和[α]-生育酚（2，000 IU/天）减缓阿尔茨海默病（AD）痴呆进展的临床试验的原理和设计。这项研究是由阿尔茨海默病合作研究（ADCS），一个积极参与AD研究的临床研究中心联盟开发的。该联盟的主要目标是设计和进行临床研究，以开发AD的治疗方法。本研究采用随机双盲、安慰剂对照、2x2析因、平行组设计，以测试两种药物治疗AD。该研究的主要结局是达到以下四个终点中任何一个的时间：死亡，机构化，三种基本日常生活活动中的两种丧失，以及临床痴呆评级（CDR）阶段从2级进展到3级。入选了中重度疾病（CDR= 2）患者，并在2年内进行了10次评价，以确定这些药物是否缩短了达到任何终点的时间。来自建立阿尔茨海默病登记研究联盟的数据库表明，有足够的把握度分析可以观察到对这一有临床意义的结局指标的治疗效果。提供了人群的招募和基线特征。选择析因设计，使用一种新的，有临床意义的终点，并选择一个队列的AD患者的中度严重程度的理由进行了讨论。
This report describes the rationale and design of a clinical trial using selegiline (10 mg/day) and [alpha]-tocopherol (2,000 IU/day) to slow the progression of dementia in Alzheimer disease (AD). This study was developed by the Alzheimer's Disease Cooperative Study (ADCS), a consortium of clinical research centers actively involved in AD research. The major goal of the consortium is to design and conduct clinical investigations leading to the development of treatments for AD. This study uses a randomized double-blind, placebo-controlled, 2x2 factorial, parallel group design to test two drugs for the treatment of AD. The primary outcome of the study is the time to reach any one of the following four endpoints: death, institutionalization, loss of two of three basic activities of daily living, and progression of Clinical Dementia Rating (CDR) stage from 2 to 3. Patients with moderately severe disease (CDR= 2) were enrolled and evaluated 10 times over a period of 2 years to determine if these agents reduce the time to reach any endpoint. A database from the Consortium to Establish a Registry for Alzheimer's Disease indicated adequate power analyses to observe a treatment effect on this clinically meaningful outcome measure. Recruitment and baseline characteristics of the population are provided. The rationale for the choice of a factorial design, the use of a novel, clinically meaningful endpoint, and the selection of a cohort of patients with AD of moderate severity are discussed.