Clinical Trials

his trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice.

This study is seeking to determine the safety and tolerability of dronabinol use in adults with SCD compared to placebo.

Do you have mitral valve disease? This clinical trial is testing different types of images taken of the heart to learn more about mitral valve disease and the best ways to treat it. You must be 60 years old to participate. The study is enrolling patients in locations in Arkansas, California, Georgia, Indiana, Louisiana, Maine, Maryland, Massachusetts, Michigan, Missouri, New York, North Carolina, Ohio, Pennsylvania, and Quebec City, Canada.

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.

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.

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 an adult who has sickle cell disease and wheezing not caused by asthma? This study will test an inhaled medicine called mometasone for people who have sickle cell disease and wheezing that is not caused by asthma. To participate in this study, you must be at least 18 years old and not have asthma, acute respiratory symptoms, or a current pain crisis. This study is located in New York, New York.

Do you or your child have a platelet, bleeding, or white blood cell disorder? This study is looking at people who have platelet or other blood disorders. The researchers will use a blood sample to look for problems with platelets and a possible genetic cause in the participant’s DNA or RNA. To participate in this study, you must be a child or adult who either is healthy or has a platelet disorder, coagulation disorder, or white blood cell disorder. This study is located in New York, New York.

Are you an adult with sickle cell disease? This study aims to determine whether monthly exchange blood transfusions (removing blood and replacing it with transfused red blood cells) can improve health and prevent or reduce the number of pain crises. To participate in this study, you must be at least 18 years old, have sickle cell disease, and have not been on a chronic exchange transfusion program for at least 2 months. This study is located in Pittsburgh, Pennsylvania.

Are you 65 years old or older, and do you have severe primary mitral regurgitation? This condition — also called degenerative mitral regurgitation — happens when there is a problem with the mitral valve itself or its supporting tissue, leading to a leaky valve. This study is comparing the safety and effectiveness of two different ways of repairing the mitral valve: transcatheter edge-to-edge repair and surgical repair. To participate in this study, your healthcare provider must determine that you are a candidate for both mitral valve transcatheter edge-to-edge repair and surgical repair. This study takes place in multiple locations across the United States and Canada.