Thompson Elizabeth J, Foote Henry P, Hill Kevin D, Hornik Christoph P
Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Duke Clinical Research Institute, Durham, NC, USA.
Contemp Clin Trials Commun. 2023 Jul 7;35:101182. doi: 10.1016/j.conctc.2023.101182. eCollection 2023 Oct.
High-quality, efficient, pharmacokinetic (PK), pharmacodynamic (PD), and safety studies in children are needed. Point-of-care trials in adults have facilitated clinical trial participation for patients and providers, minimized the disruption of clinical workflow, and capitalized on routine data collection. The feasibility and value of point-of-care trials to study PK/PD in children are unknown, but appear promising. The Opportunistic PK/PD Trial in Critically Ill Children with Heart Disease (OPTIC) is a programmatic point-of-care approach to PK/PD trials in critically ill children that seeks to overcome barriers of traditional pediatric PK/PD studies to generate safety, efficacy, PK, and PD data across multiple medications, ages, and disease processes.
This prospective, open-label, non-randomized point-of-care trial will characterize the PK/PD and safety of multiple drugs given per routine care to critically ill children with heart disease using opportunistic and scavenged biospecimen samples and data collected from the electronic health record. OPTIC has one informed consent form with drug-specific appendices, streamlining study structure and institutional review board approval. OPTIC capitalizes on routine data collection through multiple data sources that automatically capture demographics, medications, laboratory values, vital signs, flowsheets, and other clinical data. This innovative automatic data collection minimizes the burden of data collection and facilitates trial conduct. Data will be validated across sources to ensure accuracy of dataset variables.
OPTIC's point-of-care trial design and automated data acquisition via the electronic health record may provide a mechanism for conducting minimal risk, minimal burden, high efficiency trials and support drug development in historically understudied patient populations.
clinicaltrials.gov number: NCT05055830. Registered on September 24, 2021.
需要在儿童中开展高质量、高效的药代动力学（PK）、药效学（PD）和安全性研究。成人即时检验试验促进了患者和医疗服务提供者参与临床试验，将临床工作流程的干扰降至最低，并利用了常规数据收集。即时检验试验在儿童中研究PK/PD的可行性和价值尚不清楚，但似乎很有前景。危重症儿童心脏病机会性PK/PD试验（OPTIC）是一种针对危重症儿童PK/PD试验的程序性即时检验方法，旨在克服传统儿科PK/PD研究的障碍，以生成多种药物、不同年龄和疾病过程的安全性、有效性、PK和PD数据。
这项前瞻性、开放标签、非随机即时检验试验将使用机会性和采集的生物样本以及从电子健康记录中收集的数据，来描述在常规护理中给予患有心脏病的危重症儿童多种药物的PK/PD和安全性。OPTIC有一份带有特定药物附录的知情同意书，简化了研究结构并获得了机构审查委员会的批准。OPTIC通过多个数据源利用常规数据收集，这些数据源自动捕获人口统计学、药物、实验室值、生命体征、流程图和其他临床数据。这种创新的自动数据收集将数据收集负担降至最低，并便于试验开展。数据将在不同来源进行验证，以确保数据集变量的准确性。
OPTIC的即时检验试验设计和通过电子健康记录进行的自动数据采集，可能为开展低风险、低负担、高效率试验以及支持对历史上研究不足的患者群体进行药物开发提供一种机制。
clinicaltrials.gov编号：NCT05055830。于2021年9月24日注册。
