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

项目摘要/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 (现在)，这是一个紧迫的公共卫生问题。迫切需要严格、高质量的随机试验来 在这一领域发展新生儿医学的证据基础和最佳实践。作为回应，NICHD Heal Initiative NOWS药理治疗比较疗效试验(NOWS PhaCET) 联合体将设计和实施一项多中心、相对有效性、随机对照试验 (RCT)评估为临床实践指南提供信息的NOWS的最佳药物治疗。 迫切需要一个独立且经验丰富的数据协调中心(DCC)来提供 高质量和不偏不倚的生物统计专业知识，以应对 设计和进行这样一项试验：(1)建立共识，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谨可行的概念、设计和分析 随机试验(有适当的监测)；(3)开发程序和系统，以增加 提高联合体的效率，确保试验的可行性，并有效利用有限的试验参与者和 (4)确保研究设计、开发、数据收集、数据质量和数据的标准化 利用现有的医疗保健活动进行分析；以及(5)传播研究结果、公开报告和公开使用 数据。作为NICHD新生儿研究网络(NRN)的长期DCC和正在推进的 新生儿阿片类药物戒断临床试验(ACT NOW)研究，RTI成功合作 30例新生儿随机对照试验的设计与实施我们的具体目标是 NOWS PhaCET的DCC将(1)通过合作建立 就一项统一的议定书达成共识，以解决以下问题的独特可行性和执行挑战 NOWS中的药理学RCT；(2)通过灵活、高效和高质量的数据和 研究管理，促进医疗数据协调和共享；(3)保护参与者的安全和 与独立的数据安全和监测委员会(DSMC)合作研究完整性，单一IRB (4)提供及时的报告和数据分析，并与 所有财团分析和出版物的调查人员；以及(5)提供必要的后勤， 合同、通信和监管支持。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试验的各个方面都具有独特的经验，包括 神经发育随访；(2)经证实的围产期协作和科学生产力记录 研究；(3)为新生儿定制的灵活工具、流程和系统的最先进基础设施 现在纳入后续行动的区域协调小组；(4)根据需要提供多学科专家；以及(5)深度和 具有灵活的管理结构、能够快速响应不断变化的需求的专家团队。

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.