Clinical Trials

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.

This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat Severe Aplastic Anemia.

Do you have mitral valve disease? This clinical trial is testing different types of images taken of the heart to learn more about mitral valve disease and the best ways to treat it. You must be 60 years old to participate. The study is enrolling patients in locations in Arkansas, California, Georgia, Indiana, Louisiana, Maine, Maryland, Massachusetts, Michigan, Missouri, New York, North Carolina, Ohio, Pennsylvania, and Quebec City, Canada.

The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.

Are you 15 to 40 years old and have severe sickle cell disease? This study is comparing long-term outcomes for patients who receive blood and bone marrow transplants and those who receive standard treatment with medicines. Participants also have an option of contributing blood samples to be stored for future research. Participants must be 15 to 40 years old and have severe sickle cell disease. This study is located in Madison, Wisconsin.

Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.

Would you like to donate blood for a study of stored red blood cells? This study is evaluating how blood donors affect the stability of stored red blood cells. After collecting a blood sample from a healthy donor, researchers treat the red blood cells with a B vitamin called biotin and then store the sample. After about 1 week, some of the cells are transfused back into the donor to check how many of the red blood cells are still healthy after storage. After a few more weeks, more of the stored cells are transfused and tested again. To participate, your blood must meet certain requirements and you must be healthy, 18 years old or older, and weigh at least 110 pounds. This study is located in Pittsburgh, Pennsylvania.

Is your child undergoing heart surgery for congenital heart disease? This study aims to help researchers better understand the developing heart and what controls the strength of its beats by comparing it to adult hearts. Researchers will study small pieces of the heart that are removed as a normal part of surgery or repair for children with congenital heart disease. To participate in this study, your child must be undergoing cardiopulmonary bypass surgery and be 18 years old or younger. The study is located in Atlanta, Georgia.

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 65 years old or older, and do you have severe primary mitral regurgitation? This condition — also called degenerative mitral regurgitation — happens when there is a problem with the mitral valve itself or its supporting tissue, leading to a leaky valve. This study is comparing the safety and effectiveness of two different ways of repairing the mitral valve: transcatheter edge-to-edge repair and surgical repair. To participate in this study, your healthcare provider must determine that you are a candidate for both mitral valve transcatheter edge-to-edge repair and surgical repair. This study takes place in multiple locations across the United States and Canada.