BMT Clinical Trial Network at Stanford

斯坦福大学 BMT 临床试验网络

• 批准号: 10187627
• 负责人: Robert Lowsky
• 金额: $ 14.35万
• 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/10187627
• 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; Translations; Transplant Recipients; Transplantation; Transplantation Conditioning; Umbilical Cord Blood; United States National Institutes of Health; Universities; Work; active comparator; arm; base; bench to bedside; 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约 380 例成人移植手术 2016 年，包括使用匹配和异体细胞的自体和同种异体移植 不匹配的相关和无关供体、脐带血单位、离体操作的细胞产品 和转基因细胞。斯坦福大学的 BMT 项目参与基础和临床 作为单一机构以及区域、国家和国际联盟的研究。这 该部门得到美国国立卫生研究院 (NIH) 计划项目拨款的支持，超过 27 多年来对基础科学研究和临床转化试验的资助，推动了 场地。该计划通过经验丰富的生物统计学和数据管理得到加强 团体，以及符合 cGMP、FACT 和 CLIA 认证的常规细胞治疗设施 细胞加工和研究性细胞疗法的开发。该计划是一个高 应计BMT CTN中心并已招募了320名患者参加16项CTN研究。过去授予 期间，斯坦福大学研究人员担任 BMT CTN 指导委员会主席、 淋巴瘤研讨会委员会，并且是五个 CTN 委员会的成员 一个特别工作组。斯坦福大学将继续利用其计划的能力来支持 CTN的研究目标为患者推进 BMT 领域的发展是我们的共同使命 通过高质量的多中心临床试验，治疗罕见、疑难血液病。这 当前应用中提议的协议的目标是确定新策略是否可以 降低发生慢性 GVHD (cGVHD) 的风险，从而改善传统方法 同种异体骨髓移植。我们提出了一项随机 3 期临床试验，其中活性比较剂是 移植后 90 天开始使用依鲁替尼，对照组没有额外的 cGVHD 预防 战略。这项研究建立在我们之前使用该药物进行的临床前和临床工作的基础上 cGVHD 的治疗和预防。主要结果指标是类固醇的使用率 移植后 18 个月内需要 cGVHD。根据汇总数据，我们 假设依鲁替尼能够抑制 B 细胞，从而减少 cGVHD 的发展 通过不可逆抑制布鲁顿酪氨酸激酶 (BTK) 及其阻断 T 的能力来激活 通过抑制 IL-2 诱导型 T 细胞激酶 (ITK) 来激活辅助亚群。斯坦福大学骨髓移植 计划致力于继续参与 BMT CTN 试验，贡献概念 网络的考虑，以及参与网络委员会和公民活动。 ！