Clinical Trials and Studies

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.

Have you been diagnosed with sickle cell disease or another inherited hemoglobinopathy (condition that affects hemoglobin in the red blood cell)? Researchers want to know how different treatments affect how long red blood cells survive in people with hemoglobinopathies before and after treatment using labeled red blood cells. You may be able to participate if you are 18 years old or older with a diagnosis of sickle cell disease, thalassemia, or other inherited hemoglobinopathy. Individuals whose sickle cell disease or blood disorder was cured with a bone marrow transplant are also needed for the study. The study takes place in Bethesda, Maryland.

Are you pregnant and willing to donate umbilical cord blood to support future sickle cell disease therapies? This study seeks to determine the best way to collect, store, and handle blood from newborns to help researchers improve future sickle cell disease therapies. To participate in this study, you must be pregnant, 45 years old or younger, and willing to donate umbilical cord blood from your newborn. Newborns may be healthy or at risk for sickle cell disease or sickle cell trait. This study is located in Bethesda, Maryland.

Are you or your child at least 13 years old and have sickle cell disease? Do you or your child have sickle cell disease or beta-thalassemia? Researchers are testing whether a new antibody therapy prior to stem cell transplantation improves the success of the stem cell transplant in individuals at high risk for complications. Participants must be 13 years old or older. The study is taking place at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland. Patients who have the stem cell transplant must live within 1 hour of the NIH Clinical Center for 3 months after the transplant.
The stem cells for the transplant come from a fully matched donor, 4 years old or older. Donors may provide blood samples for research.

Do you or your child have sickle cell disease or sickle cell trait? This study is interested in understanding pain crises and lung complications that occur in sickle cell disease patients and patients who have other red blood cell disorders. To participate in this study, you must be at least 2 years old with known or suspected sickle cell disease, sickle cell trait, or other red blood cell disorders. Participants will have a physical exam and receive standard medical care for sickle cell disease, including routine follow-up tests and procedures. This study is located in Washington, D.C., and Bethesda, Maryland.

Are you an adult of African descent who hasn’t had a recent transfusion and doesn’t have a known pyruvate kinase deficiency? This study investigates how the PKLR gene affects the function of red blood cells and contributes to sickle cell disease. The PKLR gene helps make a protein called pyruvate kinase that is necessary for red blood cells to function properly. To participate in this study, you must be between 18 and 80 years old, with or without sickle cell disease. This study takes place in Bethesda, Maryland.

Do you or your child have sickle cell disease, and would you be willing to receive sickle cell care through telemedicine? Researchers are trying to learn more about how two different types of telemedicine can improve care for children living with sickle cell disease. Participants include caregivers (18 years old and older) who have children between 0 to 21 years old with sickle cell disease and adolescents and young adults with sickle cell disease. The study is taking place at Riley Hospital for Children Comprehensive Sickle Cell Disease Clinic in Indianapolis, Indiana.

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.

Do you have sickle cell disease and have not had a blood transfusion in the previous 8 weeks? This study will look at whether a particular gene in people who have sickle cell disease increases the risk for more severe symptoms and complications. To participate in this study, you must be between 18 and 80. This study is located in Bethesda, Maryland.

Do you have sickle cell disease and problems in your hip or shoulder joint? Researchers are studying a medicine to treat osteonecrosis (painful bone condition due to lack of blood flow) in patients with sickle cell disease. To participate in this study, you should be 18 to 80 years old and have sickle cell disease and hip or shoulder problems. This study is recruiting in Sacramento, California.