Clinical Trials Development Resource for Hematologic Disorders (U24)

描述(由申请人提供)：RTI International正在提交血液疾病临床试验开发资源(U24)的申请，RFA-HL-12-016。该资源将支持研究人员开发临床试验，这些研究人员对血液疾病的新治疗方法有想法，但无法获得设计试验以检验这些想法所需的资源。RTI资源团队包括具有不同背景的经验丰富的个人，涵盖多中心临床试验的方方面面。我们的生物统计学家在试验设计、监测和分析方面与临床研究人员进行了广泛的合作，包括血液疾病(镰状细胞病、血友病和TTP)的试验。临床研究经理将就试验实施的实际方面提出建议(招募研究地点和受试者，以及编制操作手册和其他研究材料)。该团队包括就数据输入和数据管理系统提供建议的专家程序员，具有与FDA和其他监管机构互动经验的监管专家，以及就临床试验的伦理行为提供建议的生物伦理学家。我们的团队还包括几名血液学顾问，他们在设计各种血液疾病的临床试验方面经验丰富。最后，我们的团队包括具有丰富经验的小组，提供关于试验设计和管理的正式培训。利用这些基础广泛的专业知识，RTI资源将实现两个广泛的目标。首先，资源咨询将为R34/U34调查员提供他们在试验规划阶段结束时制定高质量、有效提案所需的工具和知识。此外，这一建议将导致R34/U34调查人员制定可行的、科学合理的、设计良好的方案，这些方案可以通过R01/U01机制有效实施。我们将提供培训和指导，以提高研究人员的设计技能，以改善未来的研究，超越资源的期限。科学领导力将被授予Donald Brambilia博士作为资源首席研究员，他在领导临床试验方面拥有超过25年的统计学家经验，其中包括与国内许多领先的研究血液学家合作进行血液学研究的20年。奈杰尔·基博士是我们的首席血液学顾问，与其他5名血液学家合作支持U34/R34研究人员。我们还包括在临床试验设计的各个方面都有经验的人员， 开发、实施和运营。(摘要结束)

DESCRIPTION (provided by applicant): RTI International is submitting this application for the Clinical Trials Development Resource for Hematologic Disorders (U24), RFA-HL-12-016. The Resource will support the development of clinical trials by investigators who have ideas for new treatments of blood disorders but lack access to the resources needed to design trials to test these ideas. The RTI Resource team includes highly experienced individuals with diverse backgrounds that cover all aspects of multicenter clinical trials. Our biostatisticians have collaborated extensively with clinical investigators on trial design, monitoring, and analysis, including trials in hematological diseases (sickle cell disease, hemophilia and TTP). Clinical study managers will advise on the practical aspects of trial implementation (recruitment of study sites and subjects, and development of manuals of operations and other study materials). The team includes expert programmers to advise on data entry and data management systems, regulatory specialists with experience interacting with FDA and other regulatory agencies, and bioethicists to advise on ethical conduct of clinical trials. Our team also includes several hematology consultants experienced in designing clinical trials for a wide range of blood disorders. Finally, our team includes groups with considerable experience offering formal training on trial design and management. Drawing on this broad-based expertise, the RTI Resource will accomplish two broad Aims. First, advice from the Resource will provide the R34/U34 investigators with the tools and knowledge that they need to develop high-quality, effective proposals at the end of the trial planning phase. Also, that advice will lead to development by the R34/U34 investigators of feasible, scientifically sound, well-designed protocols that can be implemented efficiently through an R01/U01 mechanism. We will offer training and mentoring to improve the design skills of the investigators so as to improve future research studies beyond the term of the Resource. Scientific leadership will be provided Dr. Donald Brambilia as the Resource Principal Investigator who has over 25 years of experience as a statistician in leadership of clinical trials including 20 years in hematological studies working with many of the leading research hematologists in the nation. Dr. Nigel Key is our lead hematology consultant cooperating with 5 other hematologists to support the U34/R34 investigators. We also include personnel with experience in all aspects for clinical trials design, development, implementation and operations. (End of Abstract)