Faculty of Medicine, American University of Beirut, Beirut, Lebanon; Neuroscience Research Center, Faculty of Medical Sciences, Lebanese University, Beirut, Lebanon.
Faculty of Medicine, American University of Beirut, Beirut, Lebanon; Neuroscience Research Center, Faculty of Medical Sciences, Lebanese University, Beirut, Lebanon.
Glob Heart. 2019 Sep;14(3):317-325. doi: 10.1016/j.gheart.2019.07.005.
Hypertrophic cardiomyopathy (HCM) is a genetic disorder with a very large global burden for which more therapeutic management regimens are required.
In this study, the authors explore HCM-related clinical trials, determine the shortcomings leading to the lack of development of new HCM therapies, and attempt to shed light on potential areas for improvement.
In January 2019, the authors completed a search on ClinicalTrials.gov for all therapeutic and interventional clinical trials involving HCM, without any limits for location or date. Information on trial characteristics such as phase, start and end dates, sample size, experimental intervention, publications, study design, selection criteria, and results were collected and analyzed.
Sixty-three trials met the selection criteria. The average trial duration across phases was around 3 years. Around one-half of the trials were conducted in North America (United States and Canada) and 44% of the trials were in their early phases (I and II). Approximately one-third of the trials were completed. Only 14 publications were produced from all the clinical trials studied.
The study revealed a low number of trials, lack of geographic diversity, and scarcity of published results concerning HCM clinical trials. Proper management of HCM trials is of vast importance to achieve effective therapies.
肥厚型心肌病（HCM）是一种具有巨大全球负担的遗传性疾病，需要更多的治疗管理方案。
本研究作者探讨了与 HCM 相关的临床试验，确定了导致缺乏新的 HCM 治疗方法发展的不足之处，并试图阐明潜在的改进领域。
2019 年 1 月，作者在 ClinicalTrials.gov 上对所有涉及 HCM 的治疗和介入性临床试验进行了搜索，没有任何地点或日期的限制。收集并分析了试验特征（如阶段、开始和结束日期、样本量、实验干预、出版物、研究设计、选择标准和结果）的信息。
符合选择标准的有 63 项试验。各阶段的平均试验持续时间约为 3 年。大约一半的试验在北美（美国和加拿大）进行，44%的试验处于早期阶段（I 和 II 期）。大约三分之一的试验已经完成。从所有研究的临床试验中仅产生了 14 项出版物。
该研究表明，HCM 临床试验的数量较少，缺乏地域多样性，且发表的结果稀缺。适当管理 HCM 试验对于实现有效的治疗方法至关重要。
