Clinical Trials

Do you or does your child have a lipid disorder and want to improve future diagnosis and treatment? This study is exploring how different diagnostic tests can help us understand how lipid disorders, including high blood cholesterol, affect the body. Information from this study may help improve the way lipid disorders are diagnosed or treated in the future. To participate, you or your child must be at least 2 years old. The study is being conducted in Bethesda, Maryland.

Do you or your child have a lung disease? This study will investigate the genes involved in the breathing process and in the development of lung diseases such as asthma or sarcoidosis to improve understanding of the role they play. To participate in this study, you or your child must be between 2 and 90 years old. This study is located in Bethesda, Maryland, at the NIH Clinical Center.

Do you or your child have sickle cell disease and have a stem cell donor? This study is exploring how to improve bone marrow transplant procedures so the body better accepts donor stem cells. Participants in this study must be at least 4 years old. This study is located in Bethesda, Maryland.

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 15 to 40 years old and have severe sickle cell disease? This study is comparing long-term outcomes for patients who receive blood and bone marrow transplants and those who receive standard treatment with medicines. Participants also have an option of contributing blood samples to be stored for future research. Participants must be 15 to 40 years old and have severe sickle cell disease. This study is located in Madison, Wisconsin.

Is your newborn in the neonatal intensive care unit at St. Louis Children’s Hospital? This study aims to better understand breathing problems and blood flow through the heart in premature newborns to better detect conditions such as pulmonary hypertension. To participate in this study, your newborn must have been born prematurely between 24 and 29 weeks’ gestation and be a patient in the Neonatal Intensive Care Unit at St. Louis Children’s Hospital. This study is located in St. Louis, Missouri.

Are you or your child taking cyclosporine for aplastic anemia? This study is testing whether the medicine sirolimus helps prevent the return of aplastic anemia after stopping cyclosporine. Participants in this study must be 2 years or older and have severe aplastic anemia. This study is located in Bethesda, Maryland.

Have you or your child been diagnosed with aplastic anemia but not been treated yet? This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. Participants in this study must be 2 years or older and have severe aplastic anemia that has not yet been treated. This study is located in Bethesda, Maryland.

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 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.