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)

• 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
• 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

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联盟 (CPIC) 的总体目标是建立基础设施和 机构合作——重点关注罕见的先天性和围产期病毒感染——以增进了解 这些疾病，提高临床试验准备，测试疗法，推进患者护理，并最终减少 疾病负担。这些感染包括先天性巨细胞病毒 (CMV) 病、新生儿单纯疱疹 病毒（HSV）感染，以及由肠道病毒（EV）和相关人类引起的新生儿病毒性败血症 副肠病毒 (HPeV)。 由阿拉巴马大学伯明翰分校 (UAB) 的研究人员领导，并得到经验丰富的专家的支持 高效的研究团队，组成CPIC的28个研究点几十年来一直密切合作 作为抗病毒合作研究小组 (CASG)，研究罕见病的自然史和治疗 先天性和围产期传染病。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和指导的成功 职业提升活动。与所有成功的研究项目一样，关键问题的答案始终是 从而确定下一个将进一步推进该领域的重要问题。这些问题是 CPIC 研究组合的基础将促进对罕见新生儿病毒感染儿童的护理， 在我们培训下一代罕见疾病时提供体验式指导研究机会 研究人员。 CPIC 的目标是： 1）以 CASG 取得的临床研究进展为基础，进一步推进 新生儿群体中 HSV、CMV 和 EV 的诊断、治疗和了解； 2）杠杆 与 28 个 CPIC 研究中心以及利益相关者建立长期合作关系以开展临床研究 符合所有研究标准的项目； 3）通过共享推进罕见病研究 通过与数据管理和协调中心的密切合作以及通过 利用云计算能力和通用数据元素； 4）开发利用试点及可行性 提供资金以利用现有机构资源并最大限度地发挥其对科学进步的影响 与影响新生儿的罕见传染病有关； 5）支持指导研究和职业生涯 为临床研究员和初级教师提供增强机会，以促进早期阶段的发展 罕见疾病研究的研究者。我们的联盟将为罕见疾病临床带来价值 研究网络通过促进对这些疾病的独特理解，并提供经过时间考验的 研究模型和先进的药理学专业知识。