急性前循环颅内大血管闭塞血运重建临床试验设计比较研究

Clinical trial design for revascularization in acute stroke due to anterior circulation large vessel occlusion:a comparative study on 6 trials

目的通过6个比较是否用药溶栓的急性前循环颅内大血管闭塞血运重建临床试验,对临床试验设计方案和试验结果进行比较分析,以期提高临床研究者对临床试验设计重要性的全面认识。方法围绕临床试验设计的要点,针对研究人群、对照选择、主要指标、比较类型、样本含量估计等方面,分析其合理性和科学性。结果6个临床试验设计均存在一定的问题,从而导致4个试验的主要指标未达到显著性检验标准。主要问题是:研究人群与研究目标不符,采用了不合理的优效性比较类型,样本含量估计参数选取不合理,非劣效界值设定不符合最小临床重要差异等。结论临床试验设计还存在许多误区,需要进一步提高认识和重视程度。

Objective To compare and analyze clinical trial design protocols and results of 6 clinical trials concerning revascularization in acute stroke due to anterior circulation large vessel occlusion in order to improve clinical researchers’comprehensive understanding on the importance of clinical trial design.Methods As for the key points of clinical trial design,rationality and scientificity of the designs in term of study population,control selection,main indexes,comparison type and sample size estimation were analyzed.Results There were some problems in the design of 6 clinical trials.As a result,the main indexes of the 4 trials did not meet the significance test standards.The main problems were as following:study population inconsistent with the study objectives,adoption of unreasonable superiority comparison,unreasonable selection for parameters of sample size estimation,and non-inferiority margin setting not meeting the minimum clinically important difference.Conclusion There are still many misunderstandings in clinical trial design,which need further awareness and attentions.