Clinical Trials

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.

Has your child received a Fontan palliation operation? If so, they may be able to join a clinical trial that is testing whether exercise can help patients who have had this procedure. Your child must be between 8 and 19 years old, had a Fontan palliation, and can exercise. A study team member will observe the participants doing aerobic and resistance exercises live on video. The study is taking place in Palo Alto, California.

Do you want to help find new treatments for heart failure with preserved ejection fraction? This study is testing whether a medicine called spironolactone can help treat heart failure with preserved ejection fraction when paired with the standard treatment. Spironolactone helps to remove excess water and sodium from the body. To participate in this study, you must be between 50 and 99 years old and diagnosed with heart failure with preserved ejection fraction.

Do you have heart failure with preserved ejection fraction (HFPEF)? This study is testing the efficacy of a diuretic medicine called spironolactone that stops your body from absorbing too much salt and keeps your potassium levels from dropping too low. Participants of this study must be between 50 and 99 years old and already taking diuretic medicine. This study takes place in multiple locations across the United States and Sweden.

his trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice.

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.

The purpose of this study is to learn more about how the use of two different types of telemedicine (distance medical care) can address barriers to receiving comprehensive sickle cell care, and whether care can be improved.

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.

Do you have sickle cell disease or thalassemia, or are you a healthy volunteer who is willing to undergo a brain MRI? This study aims to understand whether sickle cell disease or thalassemia affects the processes that naturally increase blood flow in the brain when people experience stress. Researchers will measure blood flow using brain magnetic resonance imaging (MRI). Participants in this study must be at least 7 years old and have sickle cell disease or thalassemia. Healthy volunteers can also participate. This study is located in Los Angeles, California.

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.