BMT Clinical Trial Network at Stanford

斯坦福大学 BMT 临床试验网络

• 批准号: 10458475
• 负责人: Lori Muffly
• 金额: $ 14.01万
• 依托单位: STANFORD 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/10458475
• 关键词: Adult; Advisory Committees; Agammaglobulinaemia tyrosine kinase; Allogenic; Aplastic Anemia; Area; Autologous Transplantation; B cell repertoire; B-Cell Activation; Basic Science; Blood; Bone Marrow Transplantation; CLIA certified; Cell Count; Cell Therapy; Cells; Cities; Clinical; Clinical Research; Clinical Trials; Clinical Trials Network; Collaborations; Complication; Cyclic GMP; Data Aggregation; Development; Disease; Enrollment; Funding; Future; Goals; Graft-Versus-Tumor Induction; Grant; Hematological Disease; Hematopoietic; Homologous Transplantation; Immunoglobulins; Immunotherapy; Incidence; Institution; Institutional Review Boards; Interleukin-2; International; Intervention; Investigation; Isoantibodies; Laboratories; Lymphoma; Maintenance; Maintenance Therapy; Malignant Neoplasms; Marrow; Mediating; Medical; Mission; Morbidity - disease rate; Multi-Institutional Clinical Trial; Multicenter Trials; Multiple Myeloma; NCI Center for Cancer Research; Nature; Non-Malignant; Outcome Measure; Participant; Patient-Focused Outcomes; Patients; Phase; Phase III Clinical Trials; Pre-Clinical Model; Prevention; Prevention strategy; Procedures; Program Research Project Grants; Protocols documentation; Randomized; Reaction; Recurrent disease; Regimen; Research; Research Activity; Research Personnel; Research Support; Residual Neoplasm; Risk; Serum; Sickle Cell Anemia; Source; Steroids; T-Lymphocyte; Testing; Toxic effect; Translational Research; Transplant Recipients; Transplantation; Transplantation Conditioning; Umbilical Cord Blood; United States National Institutes of Health; Universities; Work; active comparator; arm; base; bench-to-bedside translation; chronic graft versus host disease; clinical center; comorbidity; conditioning; data management; design; disorder risk; emt protein-tyrosine kinase; experience; genetically modified cells; graft vs host disease; hematopoietic cell transplantation; immune reconstitution; improved; improved outcome; innovation; leukemia/lymphoma; member; mortality; novel; novel strategies; older patient; post-transplant; pre-clinical; prevent; primary outcome; programs; prophylactic; stem; symposium; translational clinical trial; transplant centers; trial comparing; tumor

This is a renewal application to RFA-HL-17-018 to propose that Division of Blood and Marrow Transplantation (BMT) at Stanford University continue as a Core Clinical Center for the BMT Clinical Trials Network (BMT CTN). Stanford's Program will perform about 380 adult transplants in 2016 including autologous and allogeneic transplants using cells from matched and mismatched related and unrelated donors, cord blood units, ex vivo manipulated cell products and genetically modified cells. Stanford's BMT Program participates in basic, and clinical research as a single institution, and within regional, national and international consortia. The division is supported by a National Institutes of Health (NIH) Program Project Grant with over 27 years of funding of basic scientific research, and clinical translational trials that advance the field. The Program is strengthened by highly experienced biostatics and data management groups, and a cGMP-compliant, FACT and CLIA certified Cellular Therapy Facility for routine cell processing and the development of investigational cellular therapies. The Program is a high accrual BMT CTN center and has enrolled 320 patients to 16 CTN studies. In the past granting period, Stanford investigators served as Chair of the BMT CTN Steering Committee, Chair to the Lymphoma Symposium Committee, and have been members on five CTN committees and one Task Force. Stanford will continue to leverage the capabilities of its Program to support the research goals of the CTN. It is our collective mission to advance the field of BMT for patients with rare and difficult to treat blood diseases through high quality multi-center clinical trials. The goal of the proposed protocol in the current application is to determine if a novel strategy can reduce the risk of developing chronic GVHD (cGVHD) and thereby improve upon traditional allogeneic BMT. We propose a randomized phase 3 clinical trial where the active comparator is Ibrutinib starting 90 days after transplant and the control arm is no additional cGVHD prevention strategy. This study builds upon our prior preclinical and clinical work with this agent for the treatment and prevention of cGVHD. The primary outcome measure will be the rate of steroid requiring cGVHD by 18 months after transplant. Based on an aggregate of data, we hypothesize that Ibrutinib will reduce cGVHD development due to its ability to block B cell activation via irreversible inhibition of Bruton's tyrosine kinase (BTK) and its ability to block T helper subset activation from inhibition of IL-2 inducible T cell kinase (ITK). The Stanford BMT program is committed to continued participation in BMT CTN trials, contributing concepts for consideration by the Network, and participating in Network committees and citizenship activities. !

这是RFA-HL-17-018的更新申请，建议血液和骨髓部门 斯坦福大学的移植（BMT）继续作为BMT的核心临床中心 临床试验网络（BMT CTN）。斯坦福大学的项目将进行大约380例成人移植手术 包括自体和异体移植，使用来自匹配和 不匹配的相关和无关供体，脐带血单位，体外操作细胞产品 和转基因细胞。斯坦福大学的BMT项目参与了基础和临床 研究作为一个单一的机构，并在区域，国家和国际财团。的 该部门得到了美国国立卫生研究院（NIH）计划项目赠款的支持， 多年的基础科研和临床转化试验的资助， 领域该计划得到了经验丰富的生物静力学和数据管理的加强 团体，以及符合cGMP，FACT和CLIA认证的细胞治疗设施，用于常规治疗 细胞处理和研究性细胞疗法的发展。该计划是一个高 该中心已招募了320名患者参加16项CTN研究。在过去， 在此期间，斯坦福大学的研究人员担任BMT CTN指导委员会主席， 淋巴瘤研讨会委员会，并已在五个CTN委员会的成员， 一个工作队。斯坦福大学将继续利用其计划的能力， CTN的研究目标。推进患者骨髓移植领域是我们的共同使命 通过高质量的多中心临床试验，治疗罕见和难以治疗的血液病。的 本申请中所提出的协议的目标是确定新的策略是否可以 降低发生慢性GVHD（cGVHD）的风险，从而改善传统的 同种异体骨髓移植我们提出了一项随机3期临床试验，其中活性对照药物为 移植后90天开始使用伊曲替尼，对照组不进行额外的cGVHD预防 战略这项研究建立在我们以前的临床前和临床工作与这种代理的 治疗和预防cGVHD。主要的结局指标是类固醇的使用率 移植后18个月需要cGVHD。根据数据的汇总，我们 假设伊曲替尼由于其阻断B细胞能力而将减少cGVHD的发展 通过不可逆地抑制布鲁顿酪氨酸激酶（BTK）激活和其阻断T 来自IL-2诱导型T细胞激酶（ITK）抑制的辅助细胞亚群活化。斯坦福大学BMT 该计划致力于继续参与BMT CTN试验，为 该网络的审议，并参加网络委员会和公民活动。 !