嵌合抗原受体基因修饰T细胞早期临床试验的探讨

摘要

Objective To discuss the factors that affect the design of early-phase clinical trials and analyze the designing principles and methods of early-phase clinical trials.Methods The influencing factors of the clinical trials of CAR-T cells including the product characteristics,preclinical study and safety risk were analyzed,and the important considerations for the early clinical trials were summarized in terms of study objectives,subject and trial design,etc.Results The product characteristics,pre-clinical studies and safety risks of CAR-T cells are significantly different from other drugs,all of which have significant influences on early clinical trials.Conclusion It is important to explore the effective dose,side effects,and initial effectiveness of CAR-T cells in early clinical trials,and furthermore,the clinical applicability and process stability of CAR-T cells should also be validated in early clinical trials.%目的 对影响早期临床试验设计的因素进行探讨,并深入分析早期临床试验的设计原则和方法.方法 从产品特征、临床前研究、安全性风险等方面分析CAR-T细胞临床试验的影响因素,并从试验目的、研究对象、试验设计等角度总结CAR-T细胞早期临床试验的考虑要点.结果 CAR-T细胞的产品特征、临床前研究方法和安全性风险与其它药物有显著差异,对早期临床试验有重要影响.结论 在早期临床试验中探索CAR-T细胞的有效剂量、不良反应、初步的有效性非常重要,此外,还应在早期临床试验中验证CAR-T细胞的临床可及性及工艺稳定性.