Abstract:

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.

Key words: rare diseases, clinical trials, Chinese Clinical Trial Registry, characteristic analysis