Clinical Trials and Studies

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.

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.

Are you an adult with sickle cell disease? This study aims to understand how diet and eating behaviors affect the health of people with sickle cell disease. To participate, you must be 18 years old or older. This study takes place 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.

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.

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.

Are you an adult with sickle cell disease who doesn't experience pain episodes? This study uses machine learning to identify sickle cell disease patients who may be at increased risk of chronic kidney disease. To participate in this study, you must be between 18 and 65 years old with sickle cell disease and not experiencing any painful episodes. This study takes place in Chicago, Illinois, Winston-Salem, North Carolina, and Memphis, Tennessee.