Blood and Marrow Transplant Clinical Trials Network Data Coordinating Center

血液和骨髓移植临床试验网络数据协调中心

• 批准号: 10471284
• 负责人: Mary Maresca Horowitz
• 金额: $ 713.11万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-15 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10471284
• 关键词: Address; Adherence; Aftercare; Allogenic; Area; Biological; Biological Response Modifier Therapy; Biometry; Blood; Bone Marrow Transplantation; Cell Therapy; Cells; Clinical Research; Clinical Trials; Clinical Trials Network; Collaborations; Communication; Communities; Complex; Data; Data Analyses; Data Coordinating Center; Data Management Resources; Database Management Systems; Databases; Development; Disease; Effectiveness; Enrollment; Ensure; Evaluation; Face; Funding; Funding Opportunities; Future; Geography; Glossary; Goals; Grant; Hematological Disease; Hematopoietic Stem Cell Transplantation; Information Dissemination; Institutional Review Boards; Intervention; Laboratories; Late Effects; Lead; Leadership; Logistics; Malignant - descriptor; Manuals; Marrow; Medicine; Monitor; National Cancer Institute; National Clinical Trials Network; National Heart, Lung, and Blood Institute; Nature; Non-Malignant; Outcome; Paper; Patients; Performance; Phase; Principal Investigator; Procedures; Process; Protocols documentation; Publications; Publishing; Research; Research Personnel; Resources; Risk; Role; Safety; Specimen; Structure; System; Therapy trial; Time; Transplantation; U-Series Cooperative Agreements; United States National Institutes of Health; Wisconsin; acronyms; clinical development; conditioning; curative treatments; data management; data quality; design; disorder prevention; effectiveness clinical trial; experience; follow-up; gene therapy; graft vs host disease; hematopoietic cell transplantation; improved; industry partner; innovation; international center; medical schools; meetings; member; novel; novel diagnostics; novel strategies; novel therapeutic intervention; operation; personalized medicine; preclinical development; programs; prospective; protocol development; repository; stem cell therapy; support network; therapy outcome; transplantation therapy; treatment duration

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.

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了治疗方法 恶性和非恶性血液疾病。这是一个快速发展的领域，具有活跃的临床前和临床研究 开发患者评估、细胞选择和操作以及治疗前和治疗后的新方法 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 与其他疗法相比有何作用？ HCT 和细胞治疗试验面临着独特的挑战，包括 在任何一个中心进行的手术数量相对较少，治疗适应症多种多样 需要不同的方法、干预措施的复杂性、多种并发症的风险 治疗后不久的时期，以及重要但不常见的后期影响的风险。血和 骨髓移植临床试验网络 (BMT CTN) 成立于 2001 年，旨在开发和执行 具有科学价值的前瞻性临床试验可以解决这些问题。该网络已启动 39 项试验 在数据和协调中心 (DCC) 的支持下，该中心由包括该中心在内的联盟组成 威斯康星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 支持网络并管理高效开发、实施和完成高 高质量的 I-III 期临床试验，包括概念评估和优先顺序、方案开发 适当的统计设计、及时激活和应计、安全性、合规性和数据监控 准确性以及分析和传播结果。 DCC 联盟将协调和支持所有 BMT CTN 活动维护最先进的数据库和系统，用于获取和存储 生物样本；确保数据质量、实验室合规性和监管要求； 管理合同安排和财务活动；监控和改进中心及整体网络 表现;并通过会议规划和其他后勤支持所有网络委员会和活动 支持。 DCC 还将继续通过与以下机构的合作来利用网络资源和专业知识： 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小组， NMDP 的 Be The Match BioTherapies、CIBMTR 工作委员会和干细胞治疗 结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任务至关重要 高质量和高优先级的 HCT 临床试验，特别是在非恶性血液的 HCT 领域 疾病和新型细胞/基因疗法，并且通常必须涉及非移植研究人员。