Optimal design of clinical trials for drugs designed to slow the course of Alzheimer's disease.
Optimal design of clinical trials for drugs designed to slow the course of Alzheimer's disease.
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DOI：
10.1016/j.jalz.2006.04.003
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发表时间：
2006-07-01
期刊：
影响因子：
--
通讯作者：
Carrillo, Maria C
中科院分区：
文献类型：
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作者：
Mohs, Richard C;Kawas, Claudia;Carrillo, Maria C
目前处于临床开发中的化合物被认为是通过减少淀粉样β蛋白的产生、增加清除或减少聚集来减缓阿尔茨海默病(AD)的临床进展和发病机制。在一次会议上研究了这些药物和其他药物对AD临床进展和发病机制的影响，包括：(1)用于研究治疗多发性硬化症、类风湿性关节炎、心血管疾病和骨质疏松症的疾病修饰药物的实验方法的综述；(2)对AD患者试验的可能研究设计和结果测量的讨论；以及(3)可用于AD的生物标记物的讨论。没有统一的最佳方法来研究药物对AD进展的影响，但可以指定支持延缓疾病效果的研究的特征。接受药物治疗的患者和接受安慰剂治疗的患者的相关临床结果应该在至少1年，甚至可能长达2年的时间内进行比较，同时测量反映发病机制和药物机制效应的生物标记物。
Compounds now in clinical development are hypothesized to slow the clinical progression and pathogenesis of Alzheimer's disease (AD) by their effects to diminish production, increase clearance, or decrease aggregation of amyloid beta protein. Options for investigating the effects of these and other drugs on clinical progression and pathogenesis of AD were examined at a conference that included: (1) a review of experimental methods used to investigate disease-modifying drugs for multiple sclerosis, rheumatoid arthritis, cardiovascular disease, and osteoporosis; (2) discussion of possible study designs and outcome measures for trials in patients with AD; and (3) discussion of biomarkers available for AD. There is no uniformly best way to investigate a drug's impact on AD progression but characteristics of studies supportive of a disease-slowing effect can be specified. Relevant clinical outcomes in drug-treated patients versus placebo-treated patients should be compared over at least 1 and possibly as long as 2 years with biomarkers reflective of pathogenesis and of the drug's mechanistic effects measured concurrently.