Blood and Marrow Transplant Clinical Trials Network

血液和骨髓移植临床试验网络

• 批准号: 9385494
• 负责人: JOANNE KURTZBERG
• 金额: $ 16.92万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-09-30 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9385494
• 关键词: Academic Medical Centers; Acute; Acute Graft Versus Host Disease; Address; Adoption; Adult; Allogenic; Applications Grants; Behavior Therapy; Blood; Blood Cells; Blood Platelets; Blood donor; Bone Marrow Transplantation; CD4 Lymphocyte Count; Cells; Child; Childhood; Childhood Leukemia; Chimerism; Clinical; Clinical Trials; Clinical Trials Network; Communities; Cyclophosphamide; Data; Data Analyses; Data Coordinating Center; Disease; Disease-Free Survival; Donor Selection; Dose; Engraftment; Enrollment; Foundations; Funding; Hematologic Neoplasms; Hematopoietic; Hematopoietic Stem Cell Transplantation; Hospitalization; Immune; Immunologics; Incidence; Manuscripts; Marrow; Medical center; Methods; Mission; Modeling; Multi-Institutional Clinical Trial; National Heart, Lung, and Blood Institute; Non-Malignant; Outcome; Patients; Pediatric Oncologist; Phase; Phase II Clinical Trials; Preparation; Prophylactic treatment; Protocols documentation; Randomized; Randomized Clinical Trials; Refractory; Regimen; Relapse; Reporting; Research Infrastructure; Research Personnel; Research Proposals; Residual Neoplasm; Risk; Role; Severities; Site; Source; Specimen; Standardization; Support Groups; Supportive care; T-Cell Depletion; Time; Tissues; Transplantation; Umbilical Cord Blood; Umbilical Cord Blood Transplantation; Work; arm; base; blood group; chronic graft versus host disease; conditioning; experience; fludarabine; follow-up; graft vs host disease; high risk; high risk population; in vivo; innovation; interest; leukemia; meetings; mortality; neutrophil; novel strategies; open label; pediatric patients; programs; prospective; reconstitution; success; symposium; trial comparing

Abstract The pediatric and adult transplant programs at Duke University Medical Center have served as a core clinical center for the BMT-CTN since its establishment in 2001. As a core center, Duke has made significant contributions to the network, including enrollment of 310 patients on CTN protocols, chairing two protocols (Dr. Kurtzberg chaired 0501 and Dr. Horwitz chaired 0901); serving on 6 protocol committees and 2 disease committees, authoring manuscripts and establishing and managing the immune reconstitution core for protocol 0501. The center has been in compliance with accurate and timely data and bio-specimen submission. The PI and Co-PI have regularly attended steering committee meetings, conference calls and the SSOS meetings. For this renewal of the BMT-CTN, Duke brings three specialized types of expertise to the table, (1) a long- standing and pioneering role in cord blood transplantation and banking and (2) unique experience in the treatment of pediatric patients with hematopoietic stem cell transplantation for non-malignant diseases and (3) pioneering work in cord blood and cord-tissue derived cellular therapies. In this application, Duke has added two additional centers as part of a Duke Consortium to increase participation in the network and also to increase accrual to BMT-CTN trials. The study concept proposed in this application, will serve as the foundation for a follow-on study to BMT-CTN 0501. The clinical trial proposed will compare in a randomized phase II clinical trial, the outcomes of related haplo-identical bone marrow transplantation or unrelated donor cord blood transplantation in children with high risk leukemias undergoing myeloablative conditioning therapy. The supportive care will be standardized. The preparative regimens and GvHD prophylaxis will be based on treatment `packages' well studied for the cord blood group and piloted for the haplo-BMT group. They are standardized to the extent possible, with the splitting cyclophosphamide for pre and post-transplant dosing for the haplo group and use of fludarabine in the cord blood group. The primary endpoint will be relapse-free survival at 2 years post-transplant. Overall survival, engraftment, acute and chronic Graft-versus-Host Disease, treatment related mortality, immune reconstitution, and transplant feasibility will also be explored as secondary endpoints. The predictive role of MRD at the time of transplant will also be examined to determine if either graft source offers better protection against post- transplant leukemic relapse. This study will answer critical and timely questions about the role of various alternative donors and identification of best practices for alternative donor selection for transplantation of children with hematological malignancies lacking matched related or unrelated donors. We plan to fund this trial by responding to the FOA Clinical Coordinating Center for Multi-Site Investigator- Initiated Clinical Trials (Collaborative UG3/UH3) PAR-16-300, which is due on February 13, 2017. Dr. Mary Eapen, from the CIBMTR, will serve as the PI for the grant application for the Data Coordinating Center which will utilize the infra-structure already established for the BMT-CTN. We have full support of the group at Johns Hopkins who developed the haplo post-transplant cyclophosphamide approach, The CIBMTR and the pediatric transplant community to conduct this study and prioritize accrual to the trial. Accordingly, we expect to accrue 180 patients in 3-4 years and, with two-year follow-up and data analysis, expect to complete the study in <6 years.

抽象的 杜克大学医学中心的儿科和成人移植计划已成为核心临床 自2001年成立以来，BMT-CTN中心。作为核心中心，杜克（Duke 对网络的贡献，包括在CTN方案中招收310名患者，主持了两个方案（博士 库尔茨伯格主席0501主持，霍维茨博士主持了0901）；在6个协议委员会和2个疾病中任职 委员会，创作手稿以及建立和管理协议的免疫重建核心 0501。该中心一直符合准确，及时的数据和生物特异性提交。 pi Co-Pi定期参加指导委员会会议，电话会议和SSO会议。 对于BMT-CTN的这种续约，杜克为桌子带来了三种专业的专业知识，（1） 站立和开创性的作用在脐带血移植和银行业中以及（2）独特的经验 治疗造血干细胞移植的小儿患者的非恶性疾病，（3） 脐带血和绳索派生的细胞疗法的开创性工作。在此应用程序中，杜克添加了 作为杜克大学联盟的一部分，另外两个中心，以增加网络的参与以及 增加对BMT-CTN试验的应计。 在本应用程序中提出的研究概念将成为BMT-CTN的后续研究的基础 0501。提出的临床试验将在随机II期临床试验中进行比较，相关的结果 具有单位的骨髓移植或无关的供体脐带血移植 风险白血病患有脊髓灰质治疗。支持护理将被标准化。这 制备方案和GVHD预防将基于对脐带进行良好研究的“包装” 血型并为Haplo-BMT组驾驶。他们被标准化了可能的程度 将环磷酰胺分割前和移植后的剂量，用于Haplo组，并在 脐带血组。主要终点将是移植后2年的无复发生存。总体生存， 植入，急性和慢性移植抗宿主疾病，相关死亡率，免疫重构， 并且移植可行性也将作为次要终点探索。当时MRD的预测作用 还将检查移植的移植，以确定任何一种移植物来源是否提供更好的保护 移植白血病复发。这项研究将回答有关各种作用的关键和及时的问题 替代捐助者和确定最佳实践的替代捐助者选择以移植 血液学恶性肿瘤的儿童缺乏相关或无关的捐助者。 我们计划通过响应FOA多站点研究者的FOA临床协调中心来资助这项试验。 发起的临床试验（协作UG3/UH3）PAR-16-300，该试验将于2017年2月13日到期。玛丽博士 来自CIBMTR的Eapen将作为数据协调中心赠款申请的PI 将利用已经为BMT-CTN建立的基础结构。我们在约翰斯得到了该小组的全部支持 霍普金斯（Hopkins 移植社区进行这项研究并优先考虑该试验。因此，我们希望累积 在3 - 4年内有180名患者，并进行两年的随访和数据分析，期望在<6中完成研究 年。