Clinical Trials

Have you lived in Alabama, Kentucky, Louisiana, or Mississippi for at least two years and are you interested in participating in a clinical study where researchers are trying to better understand health and disease in rural areas? The study team will track your physical activity using a provided Fitbit device and will also ask questions about your health and lifestyle through a cell phone app. You will also receive a kit to measure environmental and health factors in your homes. Participants must live in one of these counties: Dallas County, Alabama; Wilcox County, Alabama; Boyle County, Kentucky; Perry County, Kentucky; Garrard County, Kentucky; Breathitt County, Kentucky; Assumption Parish, Louisiana; Franklin Parish, Louisiana; Oktibbeha County, Mississippi; and Panola County, Mississippi.

The investigators' central hypothesis is that early combination therapy with two PAH-specific oral therapies that have been shown to be well tolerated in the pediatric population, sildenafil and bosentan, will result in better World Health Organization (WHO) functional class at 12 months after initiation of PAH treatment than therapy with sildenafil alone.

The main goal of this study is to determine whether the treatment will lead to a complete absence of severe vaso-occlusive events (VOEs) in patients with severe SCD.

Are you an adult with sickle cell disease? This study aims to determine whether monthly exchange blood transfusions (removing blood and replacing it with transfused red blood cells) can improve health and prevent or reduce the number of pain crises. To participate in this study, you must be at least 18 years old, have sickle cell disease, and have not been on a chronic exchange transfusion program for at least 2 months. This study is located in Pittsburgh, Pennsylvania.

Are you an adult who receives long-term red blood cell transfusion therapy for sickle cell disease? This 3-month study tests whether donated blood that is more than 30 days old leads to more infections in people with sickle cell disease than blood that is less than 10 days old. Some hospitals around the United States have requirements regarding the age of donor blood. This research aims to inform new evidence-based policies on the age and use of donor blood in people with sickle cell disease that rely on blood transfusions. To participate in this study, you must be between 16 and 45 years old, have sickle cell disease, and be receiving long-term red blood cell transfusion therapy. This study takes place in Atlanta, Georgia; Chapel Hill, North Carolina; and Milwaukee, Wisconsin.

Do you or your child have sickle cell disease? This study aims to develop a mathematical equation to estimate the functioning of the kidneys in children and adults with sickle cell disease. To participate in this study, you or your child must be between 5 and 50 years old. This study takes place in Birmingham, Alabama; Chicago, Illinois; and Memphis, Tennessee.

Are you or your child between the ages of 3 and 21 and experiencing pain episodes? This study tests a treatment called arginine therapy for pain episodes in children with sickle cell disease. Researchers are investigating how safe and effective this treatment is, as well as how arginine is metabolized in the body and its effects on cells. To participate in this study, you or your child must have sickle cell disease and pain that requires medical attention. This study takes place in multiple locations in the United States.