基于ClinicalTrials.gov及ChiCTR的肌萎缩侧索硬化临床试验特点分析

Analysis of clinical trial characteristics of amyotrophic lateral sclerosis based on ClinicalTrials.gov and ChiCTR

目的探讨肌萎缩侧索硬化(ALS)临床试验特点。方法全面检索美国临床试验数据库(ClinicalTrials.gov)和中国临床试验注册中心(ChiCTR)自建库至2023年12月10日期间注册的ALS临床试验相关信息,分析ALS国内外临床试验的研究设计、纳入人群、干预措施、结局评价等多方面的特点。结果ClinicalTrials.gov中ALS临床试验注册数量共765项,其中干预性研究540项;而ChiCTR中仅31项,差异较明显。美国是ALS临床试验(250项)主要开展地区,申办机构多为美国研究所或药企,北美、欧洲等地合作广泛。临床试验注册阶段多为Ⅱ期,暂停和终止的试验数量相对较少;纳入诊断标准多采用修订版EI Escorial标准;干预措施以药物治疗为主,近年研究主要围绕清除异常蛋白质、减轻神经炎症等方向展开;临床疗效评价指标以改良的肌萎缩侧索硬化功能评分量表(ALSFRS-R)运用最广泛。结论目前ALS临床试验整体数量较少,但研究数量呈上升趋势,新的干预措施不断涌现。在研究数量及创新性上国内外差异较明显,国际和区域间的合作亟需加强。

AIM To explore the characteristics of clinical trials of amyotrophic lateral sclerosis(ALS).METHODS Clinical trials of ALS were retrieved in the ClinicalTrials.gov and Chinese clinical trial registry(ChiCTR)from inception to Dec 10,2023.The data were extracted and analyzed including the registration numbers,country,trial phases,interventions,outcomes and other aspects.RESULTS A total of 765 clinical trials were registered in ClinicalTrials.gov and the interventional trials(540 items)predominated.However,only 31 items were registered in ChiCTR.The United States was the primary site for ALS clinical trials,which carried out 250 studies,and owned the most number of applicants.Moreover,broad cooperation between North America and Europe was conducted.Most of the clinical trials were phase Ⅱ.Suspended and terminated trials were relatively less.The revised EI Escorial criterion was widely used in clinical trials.Drug therapy was the main intervention measure.Recently,the research was focused on decreasing abnormal proteins and relieving the neuroinflammation.Besides,the revised ALS functional rating scale(ALSFRS-R)was the widest used index of effect evaluation.CONCLUSION At present,the total number of clinical trials of ALS is small,but it is increasing.And new treatments appear continuously.However,there are obvious differences in research quantity and innovation at home and abroad,and international and inter-regional cooperation needs to be strengthened.