Data Coordinating Center (DCC) for the Neonatal Opioid Withdrawal Syndrome Pharmacological Treatments Comparative Effectiveness Trial (NOWS PhaCET)

数据协调中心 (DCC) 新生儿阿片类药物戒断综合征药物治疗比较有效性试验 (NOWS PhaCET)

• 批准号: 10678679
• 负责人: Abhik Das
• 金额: $ 1022.6万
• 依托单位: RESEARCH TRIANGLE INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-09-20 至 2026-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10678679
• 关键词: Address; Area; Attention; Behavioral; Biometry; Caregivers; Child; Child Health; Childhood; Clinical Investigator; Clinical Practice Guideline; Clinical Trials; Clinical Trials Network; Cognitive; Collaborations; Communication; Conceptions; Consensus; Country; Data; Data Analyses; Data Collection; Data Coordinating Center; Development; Ensure; Goals; Helping to End Addiction Long-term; Hospitals; Human Resources; Informatics; Infrastructure; Institution; Institutional Review Boards; Investigational Drugs; Knowledge; Language; Leadership; Letters; Medicine; Methadone; Methodology; Monitor; Morphine; Multicenter Neonatal Research Network; National Institute of Child Health and Human Development; Neonatal; Neonatal Abstinence Syndrome; Neonatology; Newborn Infant; Office of Administrative Management; Opioid; Outcome; Participant; Perinatal; Pharmaceutical Preparations; Pharmacologic Substance; Pharmacological Treatment; Play; Population; Principal Investigator; Process; Productivity; Protocols documentation; Public Health; Publications; Publishing; Qualifying; Randomized, Controlled Trials; Reporting; Research; Research Design; Research Personnel; Resources; Risk; Role; Safety; Seizures; Site; Sleep; Standardization; Statistical Methods; Structure; Symptoms; System; Time; United States National Institutes of Health; Visual; Vomiting; Weaning; Weight Gain; clinical practice; clinical research site; comparative effectiveness; comparative effectiveness trial; data harmonization; data management; data quality; data sharing; design; evidence base; experience; feasibility trial; feeding; flexibility; follow-up; implementation barriers; multidisciplinary; neonatal care; neonate; opioid epidemic; opioid withdrawal; participant safety; pharmacologic; predictive modeling; randomized trial; real time monitoring; response; success; tool; treatment strategy; trial design

PROJECT SUMMARY/ABSTRACT Major knowledge gaps exist in optimizing pharmacologic treatment for neonatal opioid withdrawal syndrome (NOWS), a pressing public health issue. Rigorous, high quality, randomized trials are urgently needed to develop the evidence base and best practices for neonatal medicine in this area. In response, the NICHD Heal Initiative NOWS Pharmacological Treatments Comparative Effectiveness Trial (NOWS PhaCET) consortium will design and implement a multi-center, comparative effectiveness, randomized controlled trial (RCT) to assess the optimal pharmacological treatment for NOWS that informs clinical practice guidelines. There is a crucial need for an independent and experienced Data Coordinating Center (DCC) to provide high-quality and impartial biostatistical expertise to address the substantial methodologic challenges in the design and conduct of such a trial by (1) building consensus to help identify critical methodologic issues; (2) bringing objective statistical expertise to the conception, design, and analyses of a rigorous and feasible randomized trial (with appropriate monitoring); (3) developing processes and systems to increase the efficiency of the consortium, ensure trial feasibility, and effective use of limited trial participants and resources; (4) ensuring standardization of study design, development, data collection, data quality, and data analyses with existing HEAL activities; and (5) dissemination of study results, public reports, and public use data. As longtime DCC for the NICHD Neonatal Research Network (NRN) and the ongoing Advancing Clinical Trials in Neonatal Opioid Withdrawal (ACT NOW) studies, RTI has successfully collaborated in the design and implementation of 30+ RCTs in neonates informing clinical practice. Our Specific Aims as the DCC for NOWS PhaCET are to (1) Enhance the scientific rigor of the consortium by collaboratively building consensus for a uniform protocol that addresses the unique feasibility and implementation challenges of pharmacological RCTs in NOWS; (2) Optimize productivity with flexible, efficient, and high quality data and study management, and promote HEAL data harmonization and sharing; (3) Protect participant safety and study integrity working with the independent Data Safety and Monitoring Committee (DSMC), single IRB (sIRB), NICHD, and FDA (as needed); (4) Provide timely reporting and data analysis, and collaborate with investigators on all consortium analyses and publications; and (5) Provide the necessary logistical, contractual, communications, and regulatory support. The unique strengths of this application include (1) a highly qualified PI and staff uniquely experienced in all aspects of both neonatal and NOWS trials including neurodevelopmental follow-up; (2) proven track record of collaboration and scientific productivity in perinatal studies; (3) state-of-the-art infrastructure of flexible tools, processes, and systems customized for neonatal/ NOWS RCTs incorporating follow-up; (4) multidisciplinary experts available as needed; and (5) depth and breadth of expert staff with an agile administrative structure that can respond quickly to changing needs.

项目概要/摘要 在优化新生儿阿片类药物戒断综合征的药物治疗方面存在主要知识空白 （NOWS），一个紧迫的公共卫生问题。迫切需要严格、高质量、随机试验 开发该领域新生儿医学的证据基础和最佳实践。对此，NICHD Heal Initiative NOWS 药物治疗比较有效性试验 (NOWS PhaCET) 联盟将设计并实施一项多中心、比较有效性、随机对照试验 （随机对照试验）评估NOWS 的最佳药物治疗，为临床实践指南提供信息。 迫切需要一个独立且经验丰富的数据协调中心（DCC）来提供 高质量和公正的生物统计专业知识，以解决该领域的重大方法学挑战 通过以下方式设计和进行此类试验：(1) 建立共识以帮助确定关键的方法学问题； (2) 将客观的统计专业知识带入严格可行的概念、设计和分析中 随机试验（有适当的监测）； (3) 开发流程和系统以提高 联盟的效率，确保试验的可行性，并有效利用有限的试验参与者和 资源; (4) 确保研究设计、开发、数据收集、数据质量和数据的标准化 分析现有的 HEAL 活动； (5) 传播研究结果、公开报告和公众使用 数据。作为 NICHD 新生儿研究网络 (NRN) 的长期 DCC 以及正在进行的 Advancing 新生儿阿片类药物戒断 (ACT NOW) 研究的临床试验，RTI 已成功合作 设计和实施了 30 多个新生儿随机对照试验，为临床实践提供信息。我们的具体目标是 NOWS PhaCET 的 DCC 将 (1) 通过合作建设来增强联盟的科学严谨性 就统一协议达成共识，解决独特的可行性和实施挑战 NOWS 中的药理学随机对照试验； (2) 以灵活、高效、高质量的数据和数据优化生产力 研究管理，促进HEAL数据统一和共享； (3) 保护参与者的安全和 与独立数据安全和监控委员会 (DSMC)、单一 IRB 合作研究完整性 (sIRB)、NICHD 和 FDA（根据需要）； (4) 及时提供报告和数据分析，并与 所有联盟分析和出版物的调查员； (5) 提供必要的后勤保障， 合同、沟通和监管支持。该应用程序的独特优势包括 (1) 高素质的 PI 和工作人员在新生儿和 NOWS 试验的各个方面都有独特的经验，包括 神经发育随访； (2) 围产期协作和科学生产力的良好记录 研究； (3) 专为新生儿/定制的最先进的灵活工具、流程和系统基础设施 包含后续行动的NOWS 随机对照试验； （4）根据需要配备多学科专家； (5) 深度和 拥有广泛的专家团队和灵活的管理结构，可以快速响应不断变化的需求。