临床试验|概述
Our Hematopoietic Cell Transplant Program offers unique access to a range of clinical trials for children with rare or hard-to-treat conditions. Our clinicians work collaboratively with national research groups, including the Children’s Oncology Group, the Blood and Marrow Clinical Trials Network and the Pediatric Blood and Marrow Transplant Program, to develop innovations and expand transplantation for new conditions.
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最新的临床试验 |
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康格尼塔（Congenita）的疾病是一种影响人体许多部位的疾病，通常导致血液系统失败。肺部疾病，肝病和癌症是疾病和死亡的其他频繁原因。骨髓移植（BMT）可以治愈血液系统，但可以使肺和肝脏疾病和癌症的风险恶化，因为DNA受损剂（如烷基剂和辐射）通常在手术过程中使用。基于DC的生物学，我们假设有可能避免在DC患者中避免这些DNA破坏药物，并且仍然成功拥有BMT。在此方案中，我们将测试避免DNA烷基剂和辐射的方案是否可以成功地BMT，而不会损害DC患者的生存率。
NCT01659606 
Recruiting children and adults up to 65 Years (完整的资格标准） 
波士顿儿童医院 
Started: July 2012 
首席研究员：马里兰州Suneet Agarwal。博士学位, Sub-Investigator:莱斯利·莱曼（Leslie Lehmann）医学博士 
接触：马里兰州Suneet Agarwal。博士学位在617-919-7579或suneet.agarwal@childrens.harvard.edu
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急性移植与宿主疾病（AGVHD）是患有同种异体造血干细胞移植（HSCT）的最致命的并发症。AGVHD在很大程度上发生，因为骨髓移植物中的T细胞不“接受”移植受者的细胞的存在，并且会施加严重的，衰弱的且经常对受体的致命攻击，击中皮肤，肝脏，肝脏，肝脏。和胃肠道轨道，最突出。对于从无关供体的骨髓中接受骨髓的患者，AGVHD的速率可以高达80％，其中多达一半的患者死于这种并发症。尽管我们在AGVHD预防方面做出了最大的努力，但仍会发生这些严重的结果。鉴于缺乏预防当前疗法的AGVHD，迫切需要预防这种疾病的新型疗法。
NCT01012492 Recruitment status: Completed 波士顿儿童医院 上次更新发布：2019年1月21日 首席研究员：莱斯利·莱曼（Leslie Lehmann）医学博士 接触：No longer recruiting
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This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
The study examines the hypothesis that survivors of pediatric HCT are at risk for late organ toxicity and they will have identifiable biomarkers present within the first two years following HCT which will be predictive for late adverse outcomes allowing for early identification of patients at risk.
Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies Active, not recruiting Dana Farber/Boston Children's Cancer and Blood Disorders Center 开始：2015年3月 Principal Investigator:克里斯汀·邓肯（Christine Duncan），医学博士 联系人：不再招募
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这个试验将评估的有效性和安全性autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (CALD). A subject's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.
A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Active, not recruiting Dana Farber/Boston Children's Cancer and Blood Disorders Center Started: July 2013 Principal Investigator:克里斯汀·邓肯（Christine Duncan），医学博士, and大卫·威廉姆斯（David Williams），医学博士 接触：No longer recruiting
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
NCT03852498 Recruiting children or adults12 to 65 Years (完整的资格标准） Dana Farber/Boston Children's Cancer and Blood Disorders Center Started: July 2013 首席研究员：医学博士科里·卡特勒（Corey Cutler） 联系人：医学博士Corey Cutler，电话：617-851-2852或cscutler@partners.org
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这是一项前瞻性，多中心，受控的，随机的，非效率研究，可评估常规和米拉索治疗的磨牙和米拉索治疗的临床有效性，这些止血性血小板减少症的受试者预计需要具有血小板计数（S）≤10,000/μL≥2个血小板输血。
NCT02964325 Recruiting children and adults (完整的资格标准） 波士顿儿童医院 Started: Nov. 2016 Principal Investigator:Steve Sloan, MD, PhD 接触：Steve Sloan, MD, PhD，在617-355-6268
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This is a multicenter prospective collection of leftover respiratory tract secretions, paired blood and NP swabs, and clinical circumstances from pediatric HCT patients, followed by next generation genomic sequencing, transcriptome analysis, protein biomarker measurement, and statistical modeling.
NCT02926612 招募最多21岁的儿童和成人（完整的资格标准） Dana Farber/Boston Children's Cancer and Blood Disorders Center Started: June 2016 Principal Investigator:克里斯汀·邓肯（Christine Duncan），医学博士
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这项研究是为了比较除最佳支持性护理以及仅在预防肝veno-闭塞性疾病（VOD）的成人和儿科患者中，除了最佳支持性护理与最佳支持性护理外，还要比较预防性护理和最佳支持性护理的功效和安全性。或开发VOD的高风险。
NCT02851407 Recruiting children and adults 1 Month and older (完整的资格标准） Dana Farber/Boston Children's Cancer and Blood Disorders Center 开始：2016年9月 Principal Investigator:莱斯利·莱曼（Leslie E. Lehmann）医学博士
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研究人员正在研究治疗没有匹配兄弟姐妹的SCID患者的方法。目标之一是避免父母和无关人员的干细胞移植发生的问题，例如重复移植，免疫系统的不完全治愈，接触化疗以及移植物与宿主疾病。
The idea behind gene transfer is to replace the broken gene by putting a piece of genetic material (DNA) that has the normal gene into the child's cells. Gene transfer can only be done if we know which gene is missing or broken in the patient. For SCID-X1, gene transfer has been done in the laboratory and in two previous clinical trials by inserting the normal gene into stem cells from bone marrow. The bone marrow is the "factory" inside the bones that creates blood and immune cells. So fixing the gene in the bone marrow stem cells should fix the immune problem, without giving chemotherapy and without risk of graft versus host disease, because the child's own cells are used, rather than another person's.
NCT01129544 Active, not recruiting 欧宝彩票平台 开始：2010年5月 Principal Investigator:Sung-Yun Pai, MD 不招募
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The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has.
NCT03619551 Recruiting children up to 2 years (Full eligibility criteria） 欧宝彩票平台 Started: Aug. 2018 Principal Investigator:Sung-Yun Pai, MD 联系人：Sung-Yun Pai，医学博士，电话：617-919-2508或sung-yun.pai@childrens.harvard.edu