目的 分析中国临床试验注册中心（ChiCTR）儿童罕见病临床试验的注册特征与研究现状。方法 基于我国第一批和第二批罕见病目录，检索ChiCTR自建库至2025年2月7日的儿童罕见病临床试验注册信息，进行数据整理和统计分析，并对不同阶段试验特征进行比较。结果 所纳入的225项儿童罕见病注册临床试验，共涉及61个病种。其中，169项（75.11%）的组长单位位于上海市（62项）、北京市（49项）、广东省（25项）、重庆市（17项）、四川省（16项）这五个省或直辖市，169项（75.11%）组长单位为非儿童医疗机构，56项（24.89%）为儿童医疗机构。单中心研究为主（171项，76.00%），跨省多中心研究40项（17.78%），而省内多中心研究仅有10项（4.44%）。研究经费主要来源于自筹95项（42.22%）和政府支持56项（24.89%），企业出资23项（10.22%）。研究类型以探索性研究/预试验、Ⅳ期研究占56.73%，干预性研究中非随机方法最为常用（53.85%），29.33%采用药物治疗。2008—2017年实施的临床试验更倾向多中心（P=0.03）和随机化（P=0.002），2018—2025年实施的临床试验更倾向预注册（P=0.002）和设数据管理委员会（P<0.001）。结论 我国儿童罕见病研究注册数量持续增长，以药物治疗为主，预注册及数据管理受重视。一方面体现出一些地域特点，但同时也存在信息不全、方法学不严谨、资源不均等问题，建议加强临床试验全流程规范化管理，推进高质量、大规模多中心临床研究，以推动新技术和新方法在临床的科学应用。

Objective To analyze clinical trials for pediatric rare diseases registered in the Chinese Clinical Trial Registry (ChiCTR) and reveal their characteristics and development trends. Methods Based on China's first and second lists of rare diseases, pediatric rare disease clinical trials registered in ChiCTR from the establishment of the database to February 7, 2025, were retrieved. And then data organization and statistical analysis was carried out. Results A total of 225 registered clinical trials for pediatric rare diseases were included, encompassing 61 distinct disease categories. The leading units of 75.11% of the registered clinical trials were located in developed regions, and 76.00% trails were single-center studies. Fundings were primarily originated from self-raised funds and government sources (67.11%). Exploratory/pilot studies and Phase IV trials constituted 56.73% of the trails. Among interventional studies, non-randomized methods were most frequently employed (53.85%). And drug therapy was utilized in 29.33% of trials. Trials conducted between 2008-2017 were more likely to be multi-center (P=0.03) and randomized (P=0.002), while the trials conducted during 2018-2025 showed greater adoption of preregistration (P=0.002) and more tend to set up data monitoring committees (DMCs) (P<0.001). Conclusions Registered pediatric rare disease studies in China show sustained growth with getting increased attention on preregistration and data management; however, challenges still remain, such as incomplete information, methodological deficiencies, and uneven resource allocation, et al. Standardized trial management, along with high-quality and large-scale multicenter research are well recommended, which should be paid more efforts to in the future.