Virginia Commonwealth University Sickle Cell Disease

描述（由申请人提供）： VCU提议组建一个强大的团队，作为NIH镰状细胞临床研究网络的成员。所有 该小组的研究人员在镰状细胞病的管理方面具有丰富的经验。提供全面服务的成人和儿童镰状细胞临床项目已经到位。首席研究员自1986年以来几乎只照顾患有镰状细胞的成年人。共同研究者共同指导成人镰状细胞计划。本项目中列出了三名具有20年镰状细胞患者护理经验和儿科骨髓移植经验的儿科血液学家/肿瘤学家，或可用于本项目。输血医学的医学主任是该小组的一部分，以及一个重症监护和急诊科医生与休克和镰状细胞病的特殊浓度。研究人员带来了镰状细胞病合作研究、镰状细胞贫血中羟基脲多中心研究、Gardos通道抑制剂伊卡-17043 II期、II期随访、III期研究、口服铁螯合II期研究和与镰状细胞病相关的VCU休克研究的经验。除了拟议的与Zometa治疗骨痛和简单与交换输血治疗急性胸部综合征有关的试验外，可能进行的潜在试验包括全氟碳化合物试验，血液替代品（合成血红蛋白），镰状细胞疼痛的植入式药物输送装置和骨髓移植。团队拥有丰富的临床试验专业知识、新药开发专业知识和人类受试者专业知识。大约有1000名患者可参与临床试验。在过去的5年中，超过300名VCU镰状细胞患者已入组临床研究，VCU在两项镰状细胞研究中的入组人数最多或第二多。五名FTE，包括2名提供临床护理的全职镰状细胞专业护士（一名成人和一名儿科），一名全职镰状细胞专业临床协调员和两名全职镰状细胞研究协调员，可用于识别和招募患者。两项临床试验被提出：Zometa（唑来膦酸，或ZA）用于镰状细胞病的骨痛，以及一项随机试验，比较简单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。 (End摘要） 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