Zhou Yinghong, Sun Siyu, Zhang Ying, Liu Zhaoxin, Liu Chenghai, Huang Jihan
Science and Information Center, Library, Shanghai University of Traditional Chinese Medicine, Shanghai, China.
Center for Drug Clinical Research, Institute of Interdisciplinary Integrative Medicine Research, Shanghai University of Traditional Chinese Medicine, Shanghai, China.
Gastro Hep Adv. 2025 Jun 18;4(10):100725. doi: 10.1016/j.gastha.2025.100725. eCollection 2025.
This study aimed to evaluate the fundamental characteristics of clinical trials and changes over time in clinical trials of systemic therapies for hepatocellular carcinoma (HCC). Interventional clinical trials of systemic therapies registered at ClinicalTrials.gov from January 2005 to December 2024 were downloaded. Data on recruitment status, clinical phase, therapy type, trial design, outcome indicators, and other relevant factors were evaluated. A total of 1233 trials were included, of which 363(29.4%) were registered from 2005 to 2014 and 870 (70.6%) were registered from 2015 to 2024, reflecting the growing body of research on HCC. Regarding the intervention model type, single-group designs were employed in 679 (55.1%) trials, and parallel designs were employed in 489 (39.7%). A total of 209 trials were Phase 1 (17.0%), 152 (12.3%) were phase 1|phase 2, 560 were phase 2 (45.4%), 41 (3.4%) were phase 2|phase 3, 162 (13.1%) were phase 3, and 32 (2.6%) were phase 4. Small-molecule targeted agents, immune monotherapies, and targeted agent and immunotherapy combinations were the primary interventions, being used in 364 (29.5%), 434 (35.2%), and 287 (23.3) studies, respectively. Immune checkpoint inhibitors, particularly programmed death receptor 1 or programmed death ligand 1 antibodies, were the most studied immunotherapies. The development of systemic therapies for HCC have made significant progress in the past 2 decades, especially in the areas of immunotherapy and targeted therapy. The results of this study provide an important reference for the development of new HCC therapies and optimization of clinical trial design and treatment strategies.
本研究旨在评估肝细胞癌（HCC）系统治疗临床试验的基本特征以及随时间的变化。下载了2005年1月至2024年12月在ClinicalTrials.gov注册的系统治疗干预性临床试验。对招募状态、临床阶段、治疗类型、试验设计、结果指标及其他相关因素的数据进行了评估。共纳入1233项试验，其中363项（29.4%）于2005年至2014年注册，870项（70.6%）于2015年至2024年注册，这反映了对HCC研究的不断增加。关于干预模型类型，679项（55.1%）试验采用单组设计，489项（39.7%）试验采用平行设计。共有209项试验为1期（17.0%），152项（12.3%）为1期|2期，560项为2期（45.4%），41项（3.4%）为2期|3期，162项（13.1%）为3期，32项（2.6%）为4期。小分子靶向药物、免疫单药治疗以及靶向药物与免疫治疗联合是主要干预措施，分别用于364项（29.5%）、434项（35.2%）和287项（23.3%）研究中。免疫检查点抑制剂，尤其是程序性死亡受体1或程序性死亡配体1抗体，是研究最多的免疫治疗药物。在过去20年中，HCC系统治疗取得了显著进展，尤其是在免疫治疗和靶向治疗领域。本研究结果为新的HCC治疗方法的开发以及临床试验设计和治疗策略的优化提供了重要参考。
