Blood and Marrow Transplant Clinical Trials Network Data Coordinating Center

项目概要/摘要 造血干细胞移植（HCT）和细胞疗法为多种疾病提供治愈性疗法。 恶性和非恶性血液病。这是一个快速发展的领域，具有活跃的临床前和临床 开发新的方法来进行患者评估，细胞选择和操作， 治疗疗法新的诊断和治疗策略需要在明确的临床试验中进行评估， HCT与其他疗法相比的作用。HCT和细胞疗法试验面临独特的挑战，包括 在任何一个中心进行的手术数量相对较少，治疗适应症多样 需要不同的方法，干预的复杂性， 立即治疗后阶段，和重要的风险，虽然罕见，迟发效应。血液和 骨髓移植临床试验网络（BMT CTN）成立于2001年， 科学上有价值的前瞻性临床试验来解决这些问题。该网络已启动39项审判 在数据和协调中心（DCC）的支持下， 威斯康星州医学院国际血液和骨髓移植研究（CIBMTR） 国家骨髓捐赠计划和埃姆斯公司。该联盟建议继续 支持网络和管理高效率的发展，实施和完成， 高质量的I-III期临床试验，包括概念评估和优先排序， 适当的统计设计，及时激活和累积，监测安全性、合规性和数据 准确性，分析和传播结果。DCC联合体将协调和支持所有BMT CTN活动维护最先进的数据库和系统，用于获取和存储 生物标本;确保数据质量、实验室合规性和遵守法规要求; 管理合同安排和财务活动;监控和改进中心和整个网络 为所有网络委员会和活动提供会议规划和其他后勤支持 支持.发展协调委员会还将继续通过与下列机构的合作， 其他科学机构，包括国家癌症研究所资助的国家临床试验网络组， NMDP的Be The Match BioTherapies，CIBMTR的工作委员会和干细胞治疗 成果数据库，以及行业合作伙伴。这些合作对于最大限度地完成 高质量和高优先级的HCT临床试验，特别是在非恶性血液HCT领域 疾病和新的细胞/基因疗法，并且通常必须涉及非移植研究者。

Project Summary/Abstract Hematopoietic stem cell transplantation (HCT) and cellular therapy offer curative therapy for a variety of malignant and non-malignant blood diseases. This is a rapidly evolving field with active preclinical and clinical development of new approaches to patient assessment, cell selection and manipulation, and pre- and post- treatment therapy. New diagnostic and therapeutic strategies require evaluation in definitive clinical trials as does the role of HCT versus other therapies. HCT and cellular therapy trials face unique challenges, including the relatively small number of procedures performed at any single center, the diverse indications for treatment requiring dissimilar approaches, the complex nature of the interventions, the risk of multiple complications in the immediate post-treatment period, and the risk of important, though infrequent, late effects. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) was established in 2001 to develop and execute scientifically meritorious, prospective clinical trials to address these issues. The Network has launched 39 trials with the support of a Data and Coordinating Center (DCC) formed though a Consortium including the Center for International Blood and Marrow Transplant Research (CIBMTR) at the Medical College of Wisconsin, the National Marrow Donor Program, and The Emmes Corporation. This Consortium proposes to continue supporting the Network and managing the efficient development, implementation and completion of high- quality Phase I-III clinical trials, including concept evaluation and prioritization, protocol development with appropriate statistical designs, timely activation and accrual, monitoring for safety, compliance and data accuracy, and analyzing and disseminating results. The DCC Consortium will coordinate and support all BMT CTN activities maintaining a state-of-the-art database and systems for acquisition and storage of biospecimens; ensuring data quality, laboratory compliance and adherence to regulatory requirements; managing contractual arrangements and fiscal activities; monitoring and improving center and overall Network performance; and supporting all Network Committees and activities with meeting planning and other logistical support. The DCC will also continue to leverage Network resources and expertise through collaboration with other scientific bodies including the National Cancer Institute-funded National Clinical Trials Network Groups, NMDP's Be The Match BioTherapies, and CIBMTR's Working Committees and Stem Cell Therapeutic Outcomes Database, as well as industry partners. These collaborations are imperative to maximize completion of high quality and high priority HCT clinical trials, especially in the areas of HCT for non-malignant blood disorders and novel cell/gene therapies and which often must involve non-transplant investigators.