Clinical Trials

Do you or your child have severe aplastic anemia or myelodysplastic syndrome? This study aims to find new ways to make stem cell transplants safer and more effective. Researchers are testing if treating people with severe aplastic anemia with a co-infusion of blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor is safe and effective. To participate in this study, you must be between 4 and 75 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have Williams Syndrome (WS) or supravalvular aortic stenosis (SVAS) or are you interested in helping research on these conditions? The blood vessels in people with WS or SVAS have less elasticity, which can cause them to narrow. This study aims to see how blood vessel differences in people with these conditions affect organs in the body including the heart, gut, kidneys, and brain. This study is open to people with one of these conditions or healthy volunteers between 3 and 85 years old. The study takes place at the NIH Clinical Center in Bethesda,

Do you or your child have Williams-Beuren syndrome (WS) or Supravalvular Aortic Stenosis (SVAS)? Symptoms of both conditions include vascular problems that can be mild or serious. Researchers want to find out why only some people with WS and SVAS have serious symptoms while others do not. Participants in this study will provide blood or saliva samples to help researchers see what DNA or environmental changes affect the severity of the disease. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Would you like to help researchers understand how rare genetic problems may lead to cardiovascular and vascular diseases? This study will collect blood, saliva, and tissue samples to study the underlying causes of diseases. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you have bronchiectasis and get frequent lung infections? This study is exploring what causes frequent lung infections in people with bronchiectasis. Researchers will look at whether changes to a person's genes or immune system can raise the risk of these frequent infections. Participants in this study must be at least 5 years old and either healthy, diagnosed with bronchiectasis, or have a history of frequent lung infections. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Are you a healthy adult or someone with a history of lung infections such as pneumonia or bronchitis? This study aims to compare fluid and tissue samples from the nose and lungs of healthy adults with people who have a lung disease. Researchers hope to learn why some people are more susceptible to certain infections. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Are you or your child willing to donate blood or bone marrow to help research? This study collects samples of blood, bone marrow, and buccal mucosa (cells from the inside of the cheek). Researchers will use these samples to study blood cells, blood diseases, and the role of the immune system in conditions such as aplastic anemia. Participants in this study must be at least 8 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or a child in your family have a cancerous tumor or blood disease? This study will collect blood, urine, or other samples to examine for potential new or improved treatments or prevention methods. Participants in this study must be 2 years or older and either have a cancerous tumor or a cancerous or non-cancerous blood disorder, or be a compatible family member for a stem cell transplant for the patient. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have an immunodeficiency, such as fewer T cells or NK cells? You can help researchers better understand how some types of immunodeficiencies are inherited. Participants in this study must be at least 6 months of age. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have sickle cell disease and a relative willing to donate stem cells? This study is giving a second blood stem cell transplant to people with sickle cell disease who have had their disease return after one stem cell transplant. To participate in this study, you must be 4 years old or older, have sickle cell disease, and have had your disease return after receiving a blood stem cell transplant. You must also have a relative who is older than 2 years and can donate stem cells via a blood draw. This study takes place in Bethesda, Maryland.