Clinical Trials

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.

Have you been diagnosed with peripartum cardiomyopathy and are within 5 months of your due date? This study is evaluating the effectiveness of a medicine called bromocriptine to treat peripartum cardiomyopathy, or weakness of the heart muscle that occurs during or shortly after pregnancy. To participate in this study, you must be at least 18 years old, diagnosed with peripartum cardiomyopathy, and within 5 months of your delivery date. This study takes place in Pittsburgh, Pennsylvania.

Do you have overweight? Circadian rhythm disorders can cause overweight and obesity. This study is examining how not getting enough sleep causes these complications. To participate in this study, you must be between the ages of 20 and 40 and have overweight but not obesity. 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.

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 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.

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.

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.