Virginia Commonwealth University Sickle Cell Disease Clinical Network

描述(由申请人提供)： VCU建议成立一个强大的团队，作为NIH镰刀细胞临床研究网络的成员。全 该团队的研究人员在镰状细胞疾病的管理方面拥有丰富的经验。提供全方位服务的成人和儿童镰状细胞临床方案已经到位。自1986年以来，首席调查员几乎只照顾患有镰状细胞的成年人。一名联合调查员共同指导成人镰刀细胞项目。三位具有20年治疗镰状细胞患者经验和儿童骨髓移植经验的儿科血液学家/肿瘤学家被列入该项目或可用于该项目。输血医学的医疗主任是团队的一员，也是一名重症医生和急诊科医生，特别专注于休克和镰状细胞疾病。研究人员带来了来自镰状细胞病合作研究、镰状细胞性贫血中羟基脲的多中心研究、Gardos通道抑制剂ICA-17043第二阶段、第二阶段后续研究、第三阶段研究、口服铁络合第二阶段研究以及与镰状细胞疾病相关的VCU休克研究的经验。除了与Zometa治疗骨痛和单纯输血与交换输血治疗急性胸部综合征相关的拟议试验外，可能开展的试验还包括全氟碳化合物、血液替代品(合成血红蛋白)、治疗镰刀细胞疼痛的植入性药物输送装置和骨髓移植的试验。团队中有广泛的临床试验专业知识、新药开发专业知识和人体受试者专业知识。约有1000名患者可参与临床试验。在过去的5年里，超过300名VCU镰状细胞患者被纳入临床研究，在两项镰状细胞研究中，VCU一直是登记人数最多或第二多的人。有5名全职护士，包括2名提供临床护理的全职镰状细胞护士(1名成人和1名儿科)、1名全职镰状细胞临床协调员和2名部分致力于镰状细胞研究的全职协调员，可用于识别和招募病人。建议进行两项临床试验：Zometa(唑来膦酸，或Za)治疗镰状细胞病的骨痛，以及一项随机试验，比较单纯输血和交换输血治疗镰状细胞病患者急性胸部综合征的住院时间。(摘要结束) 临床中心

DESCRIPTION (provided by applicant): VCU proposes a strong team to as a member of the NIH Sickle Cell Clinical Research Network. All investigators on the team have extensive experience in the management of sickle cell disease. Full-service adult and pediatric sickle cell clinical programs are in place. The Principal Investigator has cared for adults with sickle cell almost exclusively since 1986. A Co-Investigator co-directs the Adult Sickle Cell Program. Three pediatric hematologist/oncologists with 20 years of experience caring for sickle cell patients and experience with pediatric bone marrow transplantation are listed in the project or available to the project. The medical director of Transfusion Medicine is part of the team, as well as an Intensivist and Emergency Department physician with special concentration in shock and sickle cell disease. Investigators bring experience from the Cooperative Study of Sickle Cell Disease, Multicenter Study of Hydroxyurea in Sickle Cell Anemia, Gardos Channel Inhibitor ICA-17043 Phase II, Phase II Followup, Phase III Studies, an Oral Iron Chelation Phase II Study, and VCU shock research related to sickle cell disease. Besides the proposed trials related to Zometa for bone pain and simple vs. exchange transfusion for acute chest syndrome, potential trials that could be mounted include trials of Perfluorocarbons, Blood substitutes (synthetic hemoglobins), Implantable Drug Delivery Devices for Sickle Cell Pain, and bone marrow transplantation. Extensive clinical trial expertise, new drug development expertise, and human subjects expertise are resident in the team. Approximately 1000 patients are available to approach for involvement in a clinical trial. Over 300 VCU sickle cell patients have been enrolled into clinical studies in the past 5 years, and VCU has been the top or second highest enroller in two sickle cell studies. Five FTE, including 2 full-time sickle cell-only nurse practitioners (one adult and one pediatric) giving clinical care, one full-time sickle cell-only clinical coordinator, and two full-time coordinators partly devoted to sickle cell research, are available to identify and recruit patients. Two clinical trials are proposed: Zometa (Zoledronic acid, or ZA) for Bone Pain in Sickle Cell disease, and a Randomized Trial To Compare Length Of Stay For Simple Transfusion And Exchange Transfusion For The Treatment Of Acute Chest Syndrome In Patients With Sickle Cell Disease. (End of Abstract) CLINICAL CENTER