Clinical Trials

Do you or one of your children have a congenital heart defect? This study will find both common genetic causes of congenital heart disease and ways that genes influence results of medical treatment. To participate in this study, you or your child must have congenital heart disease. This study is located in Los Angeles, Palo Alto, and San Francisco, California; New Haven, Connecticut; Boston, Massachusetts; New York and Rochester, New York; Philadelphia, Pennsylvania; and Salt Lake City, Utah.

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 or your child have pulmonary hypertension and want to help research? This study is looking to better understand pulmonary hypertension in children who have this condition. To participate in this study, you or your child must be 21 years old or younger and must have been diagnosed with pulmonary hypertension before the age of 18. This study is located at multiple sites across the United States and Canada, including California, Colorado, Massachusetts, New York, Pennsylvania, Tennessee, and Alberta, Canada.

Do you or a family member have Diamond-Blackfan anemia? This registry seeks to collect information from patients who have Diamond-Blackfan anemia (DBA), in order to help researchers and doctors learn more about the condition. The registry will also help connect patients with DBA to research and clinical studies, and possible treatment options. The registry accepts all patients diagnosed with DBA.

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.

Do you or your child have a platelet, bleeding, or white blood cell disorder? This study is looking at people who have platelet or other blood disorders. The researchers will use a blood sample to look for problems with platelets and a possible genetic cause in the participant’s DNA or RNA. To participate in this study, you must be a child or adult who either is healthy or has a platelet disorder, coagulation disorder, or white blood cell disorder. This study is located in New York, New York.

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.

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.

Do you or your child have severe asthma that is not easily controlled by inhaled corticosteroids? This study evaluates the use of five different asthma medicines to treat adults and adolescents with severe asthma. To participate in this study, you or your child must be 12 years old or older. This study takes place in multiple locations across the United States.