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.

Are you between the ages of 18 and 50 and diagnosed with Friedreich's ataxia? In this study, researchers are testing a new medication, gene therapy, to treat cardiomyopathy associated with Friedreich's ataxia, and you may be able to participate if you are not taking any corticosteroids or immunosuppressive medications to treat Friedreich's ataxia. This study is taking place in New York City, New York.

Do you have heart failure? In this clinical trial, researchers are using images of the heart and blood tests to learn more about Black and Hispanic patients with heart failure. You may be able to participate if you are at least 60 years old; of Black, Hispanic, or Caribbean origin; and have been diagnosed with heart failure. The study is taking place in locations in Connecticut, Massachusetts, and New York.

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.