Clinical Trials

Do you or your child have a heart defect that requires a procedure called extracardiac conduit–total cavopulmonary connection (EC-TCPC)? This study is evaluating the effectiveness of a new type of graft called a tissue-engineered vascular graft for EC-TPC. Participants of this study will have this procedure and several follow-up assessments with magnetic resonance imaging (MRI) to test the performance of the graft. This study takes place in Columbus, Ohio.

Does your infant have a low platelet count? This study is investigating the safety and efficacy of platelet transfusion as a treatment for infants with thrombocytopenia. Researchers are looking at the positive effects of platelet transfusion against the negative effects, such as the release of inflammatory molecules and the formation of blood clots. To participate in this study, your child must be younger than 6 months old and have a low platelet count (less than 100 × 109/L). This study takes place in Boston, Massachusetts.

Does your infant have a low platelet count? This study investigates immature platelet counts as a marker for bleeding risk in newborns with thrombocytopenia, compared with platelet counts alone. Immature platelets are the most recently produced platelets and may be a better marker of platelet production. To participate in this study, you must have a newborn who is less than 32 weeks’ gestational age, has a birth weight greater than 500 grams, and has a platelet count less than 100 × 109/L. This study takes place in multiple locations in the United States, the Netherlands, and Sweden.

Are you an adult with polycythemia vera or thrombocytosis? This study aims to find a new treatment or cure for polycythemia vera and thrombocytosis by locating genes and their changes, or mutations, that may contribute to these disorders. To participate in this study, you must have polycythemia vera with elevated hemoglobin (higher than 18 in men and 16 in women) or thrombocytosis with an elevated platelet count (higher than 450,000). This study takes place in Salt Lake City, Utah.

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.

Do you or your child have sickle cell disease and access to a smart phone? This study tests whether a mobile app can help adolescents and young adults who have sickle cell disease take medicine, such as hydroxyurea, correctly and on time. To participate in this study, you or your child must be between 12 and 21 years old, have sickle cell disease, and be currently taking hydroxyurea. This study takes place in Chicago, Illinois.

Do you or your child have sickle cell disease and receive blood transfusion treatment? This study aims to determine how long red blood cells survive in people with sickle cell disease. Some people receive blood transfusions from donor blood as a treatment for sickle cell disease. But sometimes, donor blood cells do not survive long once transfused, which can make long-term blood transfusions less effective as a treatment option. Participants in this study must be 6 years old or older and have already been receiving blood transfusion treatment for more than 3 months. This study takes place in Atlanta, Georgia.

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.

Do you have sickle cell disease and need chronic red blood cell (RBC) transfusions? This study aims to determine the effectiveness of matching donor red cells by RH genotype for people who need frequent RBC transfusions. Participants in this study must be 1 year or older. This study is located in Philadelphia, Pennsylvania.

Do you or your child have sickle cell disease? This study aims to develop a mathematical equation to estimate the functioning of the kidneys in children and adults with sickle cell disease. To participate in this study, you or your child must be between 5 and 50 years old. This study takes place in Birmingham, Alabama; Chicago, Illinois; and Memphis, Tennessee.