【摘要】 目的 系统分析2013～2025年中国降脂药物注册临床试验的纵向特征与发展趋势，为临床研究者、政策制定者及药企提供循证依据。方法 检索国家药品监督管理局药物临床试验登记与信息公示平台，提取首次公示信息日期在2013年1月1日至2025年12月31日之间的降脂药物相关临床试验，纳入适应症含“胆固醇”及“高脂血症”等关键词的试验，排除重复登记项后，对试验基本信息、药物信息及创新药试验设计等数据进行比较分析。结果 共纳入748项临床试验，其中68.45%(512项/748项)为生物等效性(BE)试验，Ⅰ、Ⅱ、Ⅲ期试验分别为125、47、64项，合计占比31.55%(236项/748项)。BE试验于2016～2019年增长，2020～2022年受疫情影响回落，2023年后再次迅猛增长;创新药临床试验数量稳步增长，2025年达峰值。94个降脂创新药涵盖前蛋白转化酶枯草溶菌素9(PCSK9)、血管生成素样蛋白3(ANGPTL3)、脂蛋白(a)[Lp(a)]、载脂蛋白C3(APOC3)等多个靶点，其中71.28%(67个/94个)由国内企业贡献，注射剂占比52.13%(49个/94个)。创新药Ⅲ期临床试验主要终点以低密度脂蛋白胆固醇(LDL-C)较基线下降百分比为主，占比65.52%(38项/58项)，硬终点主要不良心血管事件(MACE)占比15.52%(9项/58项)。靶点呈多元化拓展趋势，小干扰RNA(siRNA)等新型药物模态于2023年后爆发式增长，2025年siRNA相关试验达24项。受试者含18岁以下人群的创新药临床试验仅8项，均针对家族性高胆固醇血症适应症。结论 2013～2025年中国降脂药物临床试验实现从仿制药放量到创新药研发活跃的关键转型，靶点布局多元拓展，新型药物模态快速发展。更多还原

【Abstract】 Objective To systematically analyze the longitudinal characteristics and development trends of registered clinical trials of lipid-lowering drugs in China from 2013 to 2025, and to provide evidence-based data for clinical researchers,policy-makers,and pharmaceutical companies. Methods Data were retrieved from the National Medical Products Administration( NMPA) Clinical Trial Registration and Information Disclosure Platform. Clinical trials related to lipid-lowering drugs with the first public disclosure date ranging from January 1,2013 to December 31,2025 were included. Trials with indications containing keywords such as " cholesterol " and "hyperlipidemia " etc. were screened. After excluding duplicate registrations,comparative analyses were conducted on data regarding basic trial information,drug characteristics and the design of innovative drug trials. Results A total of 748 clinical trials were included,of which 68. 45%( 512 cases/748 cases) were bioequivalence( BE) trials,while phase Ⅰ,Ⅱ,and Ⅲ trials accounted for 125,47,and 64 trials respectively,totaling 31. 55%( 236 cases/748 cases). BE trials increased significantly from 2016 to2019,declined during 2020 to 2022 due to the COVID-19 pandemic,and surged again after 2023. The number of innovative drug trials grew steadily,peaking in 2025. Ninety-four innovative lipid-lowering drugs identified covered diverse therapeutic targets,including proprotein convertase subtilisin/kexin type 9( PCSK9),angiopoietin-like protein 3( ANGPTL3),lipoprotein( a) [Lp( a) ] and apolipoprotein C3( APOC3),with 71. 28%( 67 cases/94 cases) developed by domestic companies and injections dosage form accounting for 52. 13%( 49 cases/94 cases).The primary endpoints of phase Ⅲ trials were predominantly the percentage change in low-density lipoprotein cholesterol( LDL-C) from baseline,accounting for 65. 52%( 38 cases/58 cases),while hard endpoints such as major adverse cardiovascular events( MACE) accounted for only 15. 52%( 9 cases/58 cases). Target exploration showed a trend of diversification. Novel modalities such as small interfering RNA( siRNA) experienced explosive growth after 2023,with 24 registered trials in 2025. Only 8 innovative drug trials included subjects under 18 years old,all targeting familial hypercholesterolemia. Conclusion From 2013 to 2025,China’s lipid-lowering drug clinical trials underwent a critical transformation,shifting from generic drug expansion to active innovative drug research and development. Therapeutic targets have diversified,and novel drug modalities have developed rapidly.更多还原