慢性活动期溃疡性结肠炎非劣效临床试验设计及其定量评价

Clinical design of a non-inferiority trial and its quantitative evaluation in the patient with mild-to-moderate active ulcerative colitis

目的介绍慢性活动性轻中度溃疡性结肠炎(UC)非劣效临床试验的试验设计要点。方法根据文献和欧盟关于溃疡性结肠炎新药研究指南,对溃疡性结肠炎非劣性临床研究所涉及到的标准阳性对照、非劣界值、主要疗效指标及剂量选择等关键性问题进行设计;以一个实例进行叙述,并模拟其部分试验数据进行定量评价。结果在活动期的轻中度溃疡性结肠炎的非劣性临床研究中,阳性对照药宜选择氨基水杨酸制,剂量为3g.d-1,非劣界值应取15%,主要疗效指标评判标准采用临床疾病活动指数量表。结论本设计可为同类临床研究提供参考。

Objective To introduce the design of a non - inferiority clinical trial for evaluating the mild -to -moderate active ulcerative coli- tis. Methods Based on international literature and the European guideline on the development of new medicinal products for the treatment of ul- cerative colitis, a clinical trial was designed in involving with the positive control, non - inferiority margin, primary endpoint and the dose - finding within accepted efficacy and safety for a treatment of mild - to - moderate active ulcerative colitis. A real example of ulcerative colitis was introduced and its primary data were simulated for quantitative analysis. Result For the non - inferiority clinical trial on mild - to - moderate active ulcerative colitis, the standard positive control should be aminosalicylic acid （5 -ASA） with the dose of 3 g/day, the non -inferior margin is approximately 15% , and the CAI scale is recommended as primary endpoint. Conclusion This study can provide some important information for similar clinical research.