张冠东 杨钰 赵瑞玲

摘 要 目的：為规范我国儿科药物临床试验项目开展、促进儿科人群用药的开发与应用提供参考。方法：检索中国临床试验注册中心（Chi CTR）数据库，收集所有研究对象年龄<18周岁的药物临床试验项目，检索时限从建库起至2019年10月1日，对其项目名称、研究所涉疾病/系统、研究所处阶段、注册时间、注册状态、研究负责单位地域分布、经费来源、研究类型、是否设置数据管理委员会、征募研究对象情况、伦理委员会批准情况、是否采用盲法、是否采集人体标本及人体标本去向等信息进行统计分析。结果：共收集到儿科药物临床试验231项，共涉及21个类别的系统/疾病，包括肿瘤及瘤样病变、眼科疾病、变态反应性疾病等;研究所处阶段以上市后药物临床试验的项目有85项（占36.80%），Ⅰ～Ⅳ期药物临床试验共有77项（占33.33%）;注册项目数量逐年增加，有179项（77.49%）为预注册，52项（22.51%）为补注册;注册项目较多的前5位地区分别为北京、上海、广东、重庆、浙江，其项目数共占纳入项目总数的66.23%（153/231）;经费来源主要为自筹（57.85%）、医院资助（20.18%）、地方财政资金（10.31%）;研究类型主要为干预性研究和观察性研究，共占88.31%;有145项（占62.77%）研究暂未确定是否设置数据管理委员会;有201项（占87.01%）研究通过了相关机构的伦理委员会审查;有168项（占72.73%）研究未明确是否采用盲法;有133项（占57.58%）研究采集了人体标本，采集的人体标本去向主要为使用后销毁。结论：注册于Chi CTR的儿科药物临床试验注册数量呈逐年递增趋势，但存在地域不均衡性;我国研究者的药物临床试验注册观念正逐步建立，且已意识到数据管理对研究质量的重要性，对采集的试验标本的管理也较为规范。建议相关部门加大对临床试验注册的宣传，完善临床试验注册管理制度;相关研究者应提高临床试验注册意识，规范进行注册申报和试验项目管理。

关键词 中国临床试验注册中心;儿科人群;药物临床试验;特征

ABSTRACT   OBJECTIVE： To provide reference for standardizing pediatric clinical trials and promoting the development and application of pediatric medication in China. METHODS： Chinese Clinical Trial Registry （Chi CTR） database were searched to collect all clinical trials related to pediatric population under 18 years old from the inception to Oct. 1st，2019. Those clinical trials were analyzed statistically in respect of name of registered project， involved disease/system， research stage， registration time， registration status， regional distribution of research institutions， source of funds， research type， whether data management committee was set up，situation of recruited research objects， ethics committee approval status， whether blind method was adopted， whether to collect human specimens and whereabouts of human specimens. RESULTS： A total of 231 pediatric drug clinical trials were included. The clinical trials involved 21 kinds of diseases/systems， mainly were tumors and tumor-like lesions， ophthalmic diseases and allergic disease. The research stage has the most items （85 trials，36.80%） in post-marketing drug clinical trials; there were 77 items of phase Ⅰ-Ⅳ clinical trials （33.33%）. The number of pediatric drug clinical trials was increased year by year， and among which 179 trials were pre-registered （77.49%）， 52 trials were supplementary registration （22.51%）. Beijing， Shanghai， Guangdong， Chongqing and Zhejiang were the major regions where pediatric drug clinical trials were carried out，and the clinical trial projects of which accounted for 66.23% （153/231） of the total number of included projects. The funding sources were mainly self-financing （57.85%）， hospital finance（20.18%）， and local government finance （10.31%）. The main type of research was intervention study and observational study， accounting for 88.31% in total. Totally 145 trials （62.77%） had not yet determined whether to set up a data management committee; 201 trials （87.01%） had passed the review of the ethics committee of the relevant institution; 168 trials （72.73%） did not clarify whether the blind method was used; 133 trials （57.58%） had collected human specimens， and the collected human specimens were mainly destroyed after use. CONCLUSIONS： The number of pediatric drug clinical trials registered in Chi CTR is increasing but there is regional imbalance. Researchers have gradually established the concept of pediatric clinical trial registration， and have realized the importance of data management to research quality， and the management of collected trial specimens is relatively standardized. It is suggested that relevant departments increase the publicity of clinical trial registration and improve the clinical trial registration management system; relevant researchers should increase the awareness of clinical trial registration， standardize registration application and trial project management.

KEYWORDS   Chinese Clinical Trials Registry; Pediatric population; Drug clinical trials; Characteristics

“重视用药安全，保障儿童健康”是我国的一项长期卫生工作目标。但儿童用药品种、剂型短缺严重，世界各国普遍存在儿童超说明书用药的情况。与成人临床试验相比，儿科人群临床试验在伦理学、入组操作和评价方法等诸多方面均有明显的特殊性[1]，难以利用成人临床试验数据推导儿科人群用药的安全性和有效性，因此有必要在儿科人群中开展临床试验。

临床试验注册是医学研究伦理的需要;公开临床试验的信息，并将其置于公众监督之下，是试验研究者的义务和道德责任。临床试验注册不仅能确保追溯每项临床试验的结果，还可实现试验信息公开透明，提高研究的真实性和科学性，减少发表偏倚和不必要的重复试验，以保障公众健康和合理使用卫生资源[2-3]。药物临床试验是临床试验中的重要组成部分。目前，我国药物临床试验注册主要有两个平台——中国临床试验注册中心（Chinese Clinical Trial Registry，简称为“Chi CTR”）和药物临床试验登记与信息公示平台。其中，药物临床试验登记与信息公示平台主要登记以药品注册上市为目的的药物临床试验项目，登记的临床试验项目范围较窄;而Chi CTR网站的注册项目……