Congenital and Perinatal Infections Rare Diseases Clinical Research Consortium (RDCRC)

先天性和围产期感染罕见疾病临床研究联盟 (RDCRC)

基本信息

批准号：
10001427
负责人：
DAVID W KIMBERLIN
金额：
$ 138.39万
依托单位：
UNIVERSITY OF ALABAMA AT BIRMINGHAM
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-09-01 至 2024-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10001427
关键词：
2 year old Alabama Antiviral Agents Antiviral Therapy Area Blindness Cardiac Cessation of life Child Care Childhood Clinical Clinical Research Clinical Trials Cloud Computing Collaborations Common Data Element Communicable Diseases Communities Congenital herpes simplex Data Development Diagnosis Disease Dose Drug Kinetics Enrollment Enterovirus Exposure to Faculty Family Funding Future Future Generations Goals Health Hearing Human Industry Infection Infrastructure Lead Longitudinal Studies Mentors Modeling Morbidity - disease rate Motor Multi-site clinical study Natural History Neonatal Neonatal Mortality Neurologic Organ Outcome Parechovirus Pathogenicity Patient Care Perinatal Perinatal Infection Population Positioning Attribute Randomized Rare Diseases Readiness Recording of previous events Research Research Personnel Research Project Grants Resources Science Sepsis Septic Shock Series Simplexvirus Site Testing Therapeutic Agents Time Training United States Food and Drug Administration United States National Institutes of Health Universities Valganciclovir Viremia Virus Virus Diseases Visceral Work burden of illness career career development clinical development congenital cytomegalovirus congenital infection data management data standards experience hearing impairment improved improved outcome infancy mortality neonatal infection neonatal sepsis neonate novel therapeutics pathogenic virus pharmacometrics phase 1 study programs recruit research and development respiratory success treatment effect trial design valacyclovir

项目摘要

The overall goal of the Congenital and Perinatal Infections Consortium (CPIC) is to establish infrastructure and institutional cooperation – focusing on rare congenital and perinatal viral infections – to advance understanding of these diseases, improve clinical trial readiness, test therapies, advance patient care, and ultimately reduce disease burden. These infections include congenital cytomegalovirus (CMV) disease, neonatal herpes simplex virus (HSV) infection, and neonatal viral sepsis caused by enteroviruses (EVs) and the related human parechoviruses (HPeVs). Led by researchers at the University of Alabama at Birmingham (UAB) and supported by an experienced and highly efficient study team, the 28 study sites that will comprise the CPIC have cooperated closely for decades as the Collaborative Antiviral Study Group (CASG) to investigate the natural history and treatment of rare congenital and perinatal infectious diseases. These longstanding relationships, as well as partnerships with community and industry stakeholders, will facilitate the success of our research efforts and our mentored career enhancement activities. As with all successful research programs, answers to critical questions always lead to identification of the next important questions that will further advance the field. These questions are the basis of the CPIC research portfolio that will advance the care of children with rare neonatal viral infections and provide experiential mentored research opportunities as we train the future generation of rare diseases researchers. Aims of the CPIC are: 1) to build upon clinical research advances achieved by the CASG to further advance the diagnosis, treatment, and understanding of HSV, CMV, and EV in the neonatal population; 2) to leverage longstanding relationships across the 28 CPIC study sites and with stakeholders to conduct clinical research projects with the highest of fidelity to all research standards; 3) to advance rare diseases research by sharing standardized data through close partnership with the Data Management and Coordinating Center and by utilizing cloud computing capacities and Common Data Elements; 4) to develop and utilize pilot and feasibility funding to leverage existing institutional resources and maximize its impact on the advancement of the science related to rare infectious diseases impacting neonates; and 5) to support mentored research and career enhancement opportunities for clinical fellows and junior faculty to facilitate the development of early-stage investigators in rare diseases research. Our Consortium will bring value to the Rare Diseases Clinical Research Network by promoting unique understanding of these diseases, and by providing both a time-tested research model and advanced pharmacometric expertise.

先天性和围产期感染财团（CPIC）的总体目标是建立基础设施和机构合作 - 专注于罕见的先天性和围产期病毒感染 - 促进理解在这些疾病中，改善临床试验准备，测试疗法，预先患者护理，并最终减少伯恩疾病。这些感染包括先天性巨型病毒（CMV）疾病，新生儿疱疹病毒（HSV）感染和由肠病毒（EV）和相关人类引起的新生儿病毒败血症副病毒（HPEVS）。由阿拉巴马大学伯明翰（UAB）的研究人员领导，并在经验丰富的和高效的研究团队，将完成CPIC的28个研究站点几十年来紧密协调作为协作抗病毒研究小组（CASG），以研究罕见的自然历史和治疗先天性和围产期感染性疾病。这些长期的关系以及与社区和行业利益相关者将促进我们的研究工作的成功，我们的指导者职业增强活动。与所有成功的研究计划一样，对关键问题的答案总是导致确定将进一步推进该领域的下一个重要问题。这些问题是 CPIC研究组合的基础将推进罕见的新生儿病毒感染儿童的护理和当我们培训未来一代稀有疾病时，提供经验丰富的研究机会研究人员。 CPIC的目的是：1）在CASG取得的临床研究进步基础上以进一步促进新生儿种群中HSV，CMV和EV的诊断，治疗和理解； 2）利用在28个CPIC研究站点以及与利益相关者进行临床研究的长期关系忠于所有研究标准的项目； 3）通过共享来推进罕见疾病研究通过与数据管理和协调中心密切合作的标准化数据以及利用云计算能力和常见数据元素； 4）开发和利用飞行员和可行性资金以利用现有的机构资源并最大程度地影响其对科学发展的影响与影响新生儿的罕见传染病有关； 5）支持指导的研究和职业临床研究员和初级教师的增强机会，以促进早期发展稀有疾病研究的研究者。我们的财团将为罕见疾病带来价值通过促进对这些疾病的独特理解，并提供经过时间测试的研究网络研究模型和先进的药剂学专业知识。