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.

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.

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 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.