Sickle Cell Clinical Trial Home Page

Severe pain crises resulting from blocked blood flow, vaso-occlusion, is a common complication of sickle cell disease, often requiring hospitalization and supportive care. Patients, many of whom are children, can experience multiple crises every year, causing debilitation and cumulative tissue damage that affects life expectancy. Currently, no therapy exists that directly targets the blood blockages that cause these sickle cell crises. The Center for World Health & Medicine (CWHM) at Saint Louis University, in collaboration with Dr. William Ferguson, the Bob Costas Chair in Pediatric Medicine at Cardinal Glennon Children’s Hospital, is proposing a clinical trial to evaluate whether a marketed drug currently used in angioplasty patients can provide pain relief and shorten hospitalization of children and teenagers experiencing acute sickle cell crises. The proposed study could be conducted entirely with patients admitted to Cardinal Glennon’s Children’s Hopsital, or if sufficient funding is obtained, it could be expanded to include patients from other children’s hospitals that have collaborated with us on studies in the past, including those located in St. Louis, Kansas City, New York, and elsewhere.

CWHM scientists have expertise with particular cell surface proteins that are thought to play a significant role in the vaso-occlusion, pain, and long-term organ damage that characterize the pathology of sickle cell disease. The marketed drug of interest targets these same proteins. This clinical trial has the potential to demonstrate significant therapeutic and economic benefit (i.e. shortening the length of stay in the hospital), as well as advancing a new treatment option for physicians providing care to suffering patients. Since the drug being tested in this study is already FDA- approved and commercially available, it would have no regulatory impediment to immediate use in sickle cell disease if found to be effective.