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
