CF 3: Clinical Trials & Advocacy Core

CF-3：临床试验和宣传核心 摘要 肉瘤是一种罕见的异质性恶性肿瘤，起源于间叶细胞， 所有年龄段的人。肉瘤预示着预后不良，估计三年生存率为 转移性疾病患者为20%。肉瘤患者的治疗已经大大落后 落后于上皮和造血系统恶性肿瘤。因此肉瘤代表了 这是一个潜力未被满足的领域，其中非常需要临床前和临床进展。 肉瘤的临床研究提出了为罕见患者提供服务所固有的独特挑战 每种疾病亚型具有独特的生物学、致病性、遗传学和表观遗传学基础的人群 可以被描述和治疗利用的。肉瘤临床试验的进行是一个 需要特别关注、主动性、基础设施和专业知识的劳动密集型企业， 除了那些主要的癌症中心之外。 临床试验和倡导核心（CF-3）的主要目标是支持翻译 以及MSKCC SPORE在软组织肉瘤中的临床应用。CF-3将由关键的 纪念斯隆凯特琳癌症中心成人和儿童肉瘤项目的成员 （MSKCC），确保在所有年龄组的新疗法的流体应用。CF-3将连接 MSKCC的成人和儿童肉瘤项目;基础科学，转化和临床方面 各种SPORE项目;以及行政、生物统计和生物标本核心。 此外，CF-3将促进与其他肉瘤孢子的无缝临床合作， 临床试验的合作/政府资助机制（ETCTN/癌症治疗评价 计划[CTEP]/联盟），制药公司，倡导团体，免疫和 发展性治疗方案。CF-3将为SPORE调查人员提供以下方面的专业知识 临床试验开发和管理，临床试验支持人员， 临床试验相关管理，开展临床试验的财务支持， 相关研究以及开发和获得罕见疾病新疗法的机制。的 CF-3与其他SPORE核心密切合作，将提供急需的基础设施， 确保SPORE的翻译使命在各年龄组中及时实现， 有效和合作的方式。 合作框架3将努力促进少数群体参与者参与我们的工作， 临床研究CF-3还将制定一项计划，让患者倡导和支持团体参与进来， 我们的目标是制定临床试验方案，以最好地满足患者的需求。这一目标将 也有一个教育的组成部分，这将允许MSKCC肉瘤之间的对话， 疾病管理团队和患者，护理人员和倡导者。

CF-3: Clinical Trials and Advocacy Core ABSTRACT Sarcomas are a rare and heterogeneous group of malignancies of mesenchymal origin that affect individuals of all ages. Sarcomas portend a poor prognosis with an estimated three-year survival rate of 20% for patients with metastatic disease. Treatments for patients with sarcoma have greatly lagged behind those available for the epithelial and hematopoietic malignancies. Sarcoma therefore represents an area of unmet potential in which there is a tremendous need for preclinical and clinical advancement. Clinical research on sarcoma presents unique challenges that are inherent to serving a rare patient population with each disease subtype having a unique biologic, pathogenic, genetic, and epigenetic basis that can be characterized and therapeutically exploited. The conduct of clinical trials on sarcoma is a labor-intensive undertaking that requires specific attention, initiatives, infrastructure, and expertise, apart from and in addition to those of any major cancer center. The main objective of the Clinical Trials and Advocacy Core (CF-3) is to support the translational and clinical efforts of the MSKCC SPORE in Soft Tissue Sarcoma. The CF-3 will be jointly run by key members of the adult and pediatric sarcoma programs at Memorial Sloan Kettering Cancer Center (MSKCC), ensuring the fluid application of novel therapies across all age groups. The CF-3 will bridge the adult and pediatric sarcoma programs at MSKCC; the basic science, translational, and clinical aspects of the various SPORE projects; and the Administrative, Biostatistical, and Biospecimen Cores. Additionally, the CF-3 will facilitate seamless clinical collaboration with other Sarcoma SPOREs, cooperative/government funding mechanisms for clinical trials (ETCTN/Cancer Therapy Evaluation Program [CTEP]/Alliance), pharmaceutical companies, advocacy groups, and immune and developmental therapeutic programs. The CF-3 will provide SPORE investigators with expertise in clinical trial development and administration, support staff for the clinical trials, expertise in and administration of clinical trial correlates, financial support for conducting clinical trials and performing correlative studies, and mechanisms for developing and acquiring novel therapies for rare diseases. The CF-3, in close collaboration with the other SPORE cores, will provide the much-needed infrastructure to ensure that the translational mission of the SPORE is realized across age groups in a timely, cost- effective, and cooperative manner. CF-3 will work to facilitate the engagement and involvement of minority participants in our clinical research. CF-3 will also develop a program to involve patient advocacy and support groups, with the goal of developing our clinical trial protocols to best serve the needs of the patients. This aim will also have an educational component, which will allow for conversations between the MSKCC Sarcoma Disease Management Team and patients, caregivers, and advocates.