BPCA-PTN COVID-19 STUDIES

BPCA-PTN COVID-19 研究

• 批准号: 10267885
• 负责人: KANECIA ZIMMERMAN, MD
• 金额: $ 500万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-21 至 2023-09-20
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10267885
• 关键词: 2019-nCoV; Academia; Affect; Best Pharmaceuticals for Children Act; Biological; Biological Markers; COVID-19; COVID-19 pandemic; Centers for Disease Control and Prevention (U.S.); Cessation of life; Child; Childhood; China; Clinical; Clinical Data; Clinical Trials Design; Communities; Coronavirus; Country; Data; Data Coordinating Center; Diagnosis; Disease; Disease Outbreaks; Dose; Drug Industry; Drug Kinetics; Effectiveness; Epidemiology; Exposure to; Extramural Activities; Failure; Funding; Future; Infant; Infection; Inflammatory; Institution; Intervention; Knowledge; Legal patent; Life; Names; National Institute of Child Health and Human Development; Network Infrastructure; Outcome; Patient Care; Patients; Pharmaceutical Preparations; Pneumonia; Population; Prevention; Preventive; Procedures; Provider; Public Health; Recommendation; Reporting; Research; Risk; Risk Factors; Safety; Sampling; Science; Sepsis; Symptoms; Syndrome; System; Therapeutic; United States Food and Drug Administration; United States National Institutes of Health; Work; clinically relevant; coronavirus disease; data harmonization; design; disease diagnosis; drug development; improved; infection rate; interest; novel; off-patent; pandemic disease; phase 1 designs; programs; sample collection; standard of care

For the last 25 years the scientific community, including academia, the National Institutes of Health (NIH), the Food and Drug Administration (FDA) and the pharmaceutical industry have worked to improve the knowledge of medications used in children. The responsibility for the implementation and oversight for improving drug development has been delegated primarily to the FDA (for on-patent drugs) and to the NIH (for off-patent drugs). The Pediatric Trials Network (PTN) has been established and funded under the Best Pharmaceuticals for Children Act (BPCA) Clinical Program implemented by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Many PTN studies historically capitalize on standard of care (SOC) procedures, such as data and biological-sample collection from infants and children already receiving drugs of interest (DOIs), and have produced meaningful pharmacokinetic (PK) data resulting in improved dosing recommendations in infants and children. These studies do not prescribe or administer drugs to children, but rather collected samples from children who were already receiving drugs per SOC as prescribed by their treating provider. In addition, preliminary data obtained through opportunistic studies have served to design phase 1 through 3 trials in children as well as support applications for extramural research. The coronavirus disease 2019 (COVID-19) pandemic is caused by the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The infection may range from the patient having no symptoms to causing a wide range of symptoms, such as mild cold like symptom to life-threatening pneumonias, sepsis, and multi-organ system failure. COVID-19 first emerged in December 2019 when a cluster of patients in Wuhan, China developed severe pneumonias of unknown cause. [Wiersinga et al, JAMA doi:10.1001/jama2020.12839]. As of August 11, 2020, SARS-CoV-2 has affected more than 200 countries, resulting in 20,124,437 million identified cases with over 737K confirmed deaths [https://www.cdc.gov/coronavirus/2019-ncov]. In April 2020 at the peak of the COVID-19 outbreak, there was recognized a new onset of hyperinflammatory symptoms in children who either had active disease or in children who had been previously exposed to COVID-19 with or without symptoms. This syndrome was named multisystem inflammatory syndrome in children (MIS-C) by the CDC in May 2020. As of July 29, 2020, 570 patients have met the criteria for MIS-C in the US [https://www.cdc.gov/mmwr/volumes/69/wr/mm6932e2.htm]. As the pandemic continues to spread throughout the US and the world, the scientific community anticipates that the number of children infected and affected will continue to rise. There must be an organized concerted collaborative effort between public health, patient care, and research in order for the appropriate prevention, identification and treatment of children impacted by COVID-19. Since COVID-19 has ravaged the world with inordinate high infection rates and death, the need for preventive, research and clinical data have become paramount and top priority for NIH as a research and funding institution. Furthermore, NIH, academia, clinicians, and others in the scientific community are concluding that the harmonization of all epidemiology and clinical related data as well as clinically relevant outcomes vital to identification and treatment of patients at-risk for COVID-related illnesses, such as the multi-system inflammatory syndrome in children (MIS-C), are critical to successfully managing the pandemic. These COVID-19 related studies will leverage the expertise and existing data within the PTN infrastructure of the BPCA program (BPCA PTN and Data Coordinating Center) to expand the research on SARS-CoV-2 (COVID-19) in children.

在过去的25年里，科学界，包括学术界，国立科学院 美国国家卫生研究院(NIH)、食品和药物管理局(FDA)和制药业已经努力 提高儿童用药知识。落实的责任 改进药物开发的监督主要委托给FDA(针对专利 药物)和国家卫生研究院(用于非专利药物)。儿科试验网络(PTN)已经 根据儿童最佳药品法案(BPCA)临床计划建立和资助 由尤尼斯·肯尼迪·施莱弗国家儿童健康和人类发展研究所实施 (NICHD)。 许多PTN研究历来利用标准护理(SOC)程序，如数据和 从已经接受感兴趣药物(DOI)的婴儿和儿童采集生物样本， 并产生了有意义的药代动力学(PK)数据，从而改进了剂量建议 在婴儿和儿童身上。这些研究不给儿童开处方或给他们用药，而是 从已经按照他们的处方按照SOC接受药物治疗的儿童身上收集样本 治疗提供者。此外，通过机会主义研究获得的初步数据也起到了作用 设计1至3期儿童试验，并支持校外应用程序 研究。 冠状病毒病2019年(新冠肺炎)大流行是由新型严重急性呼吸道综合征引起的 SARS冠状病毒2型(SARS-CoV-2)。感染的范围从没有症状的患者到 引起一系列症状，如轻度感冒样症状到危及生命的肺炎， 败血症和多器官系统衰竭。新冠肺炎最早出现在2019年12月，当时一群 在武汉的患者中，中国患上了原因不明的重症肺炎。[Wiersinga等人，JAMA DOI：10.1001/JAMA2020.12839]。截至2020年8月11日，SARS-CoV-2已感染200多人 导致201.244.37亿例确诊病例，确认死亡人数超过73.7万人 [https://www.cdc.gov/coronavirus/2019-ncov].2020年4月，也就是新冠肺炎爆发的高峰期， 在活动性强的儿童中，新的炎症性症状的发作被认为是新的。 无论有无症状，以前接触过新冠肺炎的儿童都有可能感染这种疾病。这 该综合征于2020年5月被美国疾病控制与预防中心命名为儿童多系统炎症综合征(MIS-C)。AS 截至2020年7月29日，美国已有570名患者符合MI-C标准 [https://www.cdc.gov/mmwr/volumes/69/wr/mm6932e2.htm].随着大流行的继续蔓延 在美国和世界各地，科学界预计，儿童的数量 感染和受影响的人数将继续上升。必须有一个有组织的协调一致的合作 公共卫生、病人护理和研究之间的努力，以便进行适当的预防， 新冠肺炎影响儿童的识别和治疗。 由于新冠肺炎已经以过高的感染率和死亡率肆虐世界，因此需要 对于预防，研究和临床数据已经成为NIH作为 研究和资助机构。此外，美国国立卫生研究院、学术界、临床医生和其他 科学界的结论是，所有流行病学和临床相关因素的协调 对识别和治疗患者至关重要的数据和临床相关结果 COVID相关疾病的高危人群，如儿童的多系统炎症综合征 (MIS-C)是成功控制这一大流行的关键。 这些与新冠肺炎相关的研究将利用PTN内的专业知识和现有数据 BPCA方案的基础设施(BPCA PTN和数据协调中心)，以扩大 儿童SARS-CoV-2(新冠肺炎)感染的研究