Blood & Marrow Clinical Trials Network - Hutchinson Center Core Clinical Sit

描述(申请人提供)：造血干细胞(HCT)移植为各种恶性和非恶性疾病提供了根治疗法。它受到供者可获得性、移植相关毒性、移植物抗宿主病(GVHD)、恶性复发、感染以及对一些患者移植后生活质量下降的限制。血液和骨髓移植临床试验网络(BMT CTN)继续在前一个资助期取得进展，建议开发和执行科学上有价值的、前瞻性的临床试验，以解决HCT的关键问题。由于儿科移植的复杂性，特别是那些患有罕见遗传性疾病的患者，这是复杂和难以研究的，我们将特别关注并集中科学专业知识，利用该网络的协调力量来改善这一独特人群的骨髓移植结果。BMT CTN还将通过合作研究和对生物终点的辅助研究来扩大和利用网络资源，这些生物终点将与临床终点整合并补充。该网络还将寻求与其他科学机构的积极合作，包括NCI资助的癌症合作小组，以提高临床移植研究的效率，为可能从骨髓移植中受益的大量患者提供帮助，并最大限度地成功完成高质量和高优先级的临床试验。弗雷德·哈钦森癌症研究中心是进行多中心第二阶段试验的理想场所，更重要的是，在六个关键领域进行预期第三阶段试验，包括：替代供体和移植物来源；与方案相关的毒性；移植物抗宿主病；疾病复发；感染和免疫重建；以及后期效应和生活质量。该中心致力于合作的临床研究，可以提供一个出色的环境，在其中进行具有最先进的临床和实验室设施的各种研究。相关性(参见说明书)：HSCT临床研究的主要障碍包括条件的异质性、任何一个中心有HSCT适应症的人数很少、不同中心的治疗策略不同，以及缺乏协调国家合作努力的资源。血液和骨髓临床试验网络使用多中心方法，在罕见疾病的患者中有效地进行新疗法的临床试验。

DESCRIPTION (provided by applicant): Hematopoietic stem cell (HCT) transplantation offers curative therapy for a variety of malignant and non- malignant disorders. It is limited by donor availability, transplant related toxicity, graft vs. host disease (GVHD), malignant relapse, infections, and for some patients, reduction in their post-transplant quality of life. Continuing progress from the previous funding period, the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) proposes to develop and execute scientifically meritorious, prospective clinical trials addressing key issues in HCT. Due to the complexity of pediatric transplantation, especially in those with rare inherited disorders, which are complex and difficult to study, we will devote special attention and focused scientific expertise to using the coordinated strength of the Network to improve BMT outcomes for this unique population. The BMT CTN will also amplify and leverage Network resources through collaborative research and ancillary studies of biologic endpoints that will be integrated with and complement clinical endpoints. The Network will also seek active collaboration with other scientific bodies including NCI- funded Cancer Cooperative Groups to improve the efficiency of clinical transplant studies for the large number of patients who could benefit from BMT and to maximize our successful completion of high quality and high priority clinical trials. The Fred Hutchinson Cancer Research Center is an ideal site to conduct multicenter Phase II and more importantly, prospective Phase III trials in six key areas including: alternative donors and graft sources; regimen related toxicity; GVHD; disease recurrence; infection and immune reconstitution; and late effects and quality of life. The Center is deeply committed to cooperative clinical research and can provide an outstanding environment in which to conduct the sorts of studies described with state-of-the-art clinical and laboratory facilities. RELEVANCE (See instructions): The major barriers to clinical studies in HSCT include the heterogeneity of conditions, the small numbers of individuals with an indication for HSCT at any one center, the differences in treatment strategies at various centers, and the lack of resources to coordinate national collaborative efforts. The Blood & Marrow Clinical Trial Network uses a multicenter approach to effectively conduct clinical trials of novel therapies in patients with uncommon conditions.