采用导入期设计提高肌萎缩侧索硬化症临床试验的效率。
Improving efficiency of ALS clinical trials using lead-in designs.
作者信息
Moore Dan H, Miller Robert G
机构信息
Research Institute CPMC, San Francisco, CA 94115, USA.
出版信息
Amyotroph Lateral Scler Other Motor Neuron Disord. 2004 Sep;5 Suppl 1:57-60. doi: 10.1080/17434470410019997.
We describe designs for clinical trials in ALS including two that are more efficient than the standard two-arm, parallel design. The more efficient designs incorporate a lead-in period followed by a randomized intervention (drug or placebo) period. Efficacy of the more efficient designs is based on measuring, within each patient, the difference in slope while on the new treatment compared to the lead-in period. We demonstrate, with sample size calculations, that the lead-in designs are considerably more efficient than the standard two-arm, parallel design. Sample sizes can be reduced by 44% for a 12-month study using ALSFRS rate of decline as a primary endpoint for a two-arm trial with 4 months lead-in compared to a parallel design. A sample size reduction of 70% can be realized with variable lead-in compared to a parallel design.
我们描述了肌萎缩侧索硬化症（ALS）临床试验的设计，其中包括两种比标准双臂平行设计更有效的设计。更有效的设计包括一个导入期，随后是随机干预（药物或安慰剂）期。更有效设计的疗效基于测量每个患者在接受新治疗时与导入期相比斜率的差异。通过样本量计算，我们证明导入期设计比标准双臂平行设计效率高得多。对于一项为期12个月的研究，以ALSFRS下降率作为主要终点，与平行设计相比，双臂试验采用4个月导入期时样本量可减少44%。与平行设计相比，采用可变导入期可实现70%的样本量减少。
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