Clinical Trials

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.

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.

Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.

Are you or your child at least 13 years old and have sickle cell disease? In this stidy, researchers are trying to find out if a new medication improves the success of a blood stem cell transplant. People 13 years old or older who are eligible for a blood stem cell transplant to treat sickle cell disease may be able to participate. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood for the study. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.

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.

Do you or your child need another blood stem cell transplant for sickle cell disease? This study will give a second transplant to people who have sickle cell disease and whose disease has returned but still have some donor cells in their body. Participants in this study must be at least 4 years old and had a transplant but the disease returned, and their donor relatives. This study is located 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.