Clinical Trials and Studies

Have you been diagnosed with a bone marrow failure disease (BMFD)? Researchers are testing whether adding treosulfan to a conditioning regimen results in patients having less severe complications after a blood or bone marrow transplant. To participate in this study, you or your child should be 1 to 49 years old and have BMFD. This study is recruiting in multiple locations.

This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat Severe Aplastic Anemia.

Are you at least 18 years old with chronic anemia? Researchers in this study are trying to find a better treatment for chronic anemia and are testing a new medication to see if it works well. To participate, you must be at least 18 years old and have chronic anemia. This study is taking place at the National Institutes of Health Clinical Center.

Are you aged 18 and older with an immune bone marrow failure? This study is testing a drug called ruxolitinib in people with different types of immune bone marrow failure. Participants will have blood tests every week while they are taking the drug. These tests can be done by the participant s own physician and the results sent to the researchers. This study will take place at the National Institutes of Health Clinical Center 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.

Do you have a baby with sickle cell disease? This study is evaluating how sickle cell disease may affect the development of babies, and if support to the caregiver can influence the baby’s level of development. To participate in this study, your child should be 6 months to 31 months old and have sickle cell disease. This study is in Saint Louis, Missouri.

Are you considering a blood stem cell transplant? This study is investigating a new method for collecting blood stem cells from donors to see if it reduces transplant complications, such as rejection, in patients who have blood diseases. To participate in this study, you must have a well-matched donor; you and your donor must be between 4 and 80 years old; and you must not be a candidate for immunosuppressive therapy. This study is located in Bethesda, Maryland.

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.

Do you have short telomeres and a telomere gene mutation? Some people who have aplastic anemia have very short telomeres, which protect the ends of DNA in chromosomes. This study is testing whether low doses of the medicine danazol help prevent telomeres from getting shorter and reduce signs of damage from aplastic anemia or related conditions. Participants in this study must be 3 years or older and have a telomere disease and signs of aplastic anemia, lung disease such as pulmonary fibrosis, or liver disease. This study is located in Bethesda, Maryland.

Have you received a stem cell transplant? This study aims to monitor the long-term health of patients who have received a donor stem cell transplant from the NIH Clinical Center. To participate in this study, you must be between 10 and 80 years old and had your transplant more than 3 years ago. This study is located in Bethesda, Maryland.