Clinical Trials

The investigators' central hypothesis is that early combination therapy with two PAH-specific oral therapies that have been shown to be well tolerated in the pediatric population, sildenafil and bosentan, will result in better World Health Organization (WHO) functional class at 12 months after initiation of PAH treatment than therapy with sildenafil alone.

Have you or your child been diagnosed with a blood disorder? If so, you (or they) may be able to participate in a clinical study where researchers are studying a type of infusion using cord blood. Participants can be of any age but must have a blood disorder diagnosed by their doctor. The study is taking place in locations in Alabama, Arizona, California, Colorado, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Maryland, Massachusetts, Michigan, Minnesota, Mississippi, Missouri, Nebraska, New York, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Virginia, Washington, Washington, D.C., West Virginia, and Wisconsin.

Do you know someone who will be having heart surgery? This study aims to identify what genetic patterns might cause atrial fibrillation and other adverse events that happen after heart surgery. To participate in this study, you must be between 20 and 90 years old. The study is in three locations in Massachusetts and Texas.

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 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 between 40 and 85 with worsening idiopathic pulmonary fibrosis? This study aims to test a new combination treatment for acute exacerbations, a life-threatening complication of idiopathic pulmonary fibrosis (IPF) that has no approved treatment. The treatment is meant to help stop part of the immune system from further injuring the lung tissue. To participate in this study, you must be between 40 and 85 years old and have worsening IPF. This study is located in Birmingham, Alabama; Boston, Massachusetts; Philadelphia, Pennsylvania; and Pittsburgh, Pennsylvania.

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.

Do you want to help find new treatments for heart failure with preserved ejection fraction? This study is testing whether a medicine called spironolactone can help treat heart failure with preserved ejection fraction when paired with the standard treatment. Spironolactone helps to remove excess water and sodium from the body. To participate in this study, you must be between 50 and 99 years old and diagnosed with heart failure with preserved ejection fraction.

Does your child have higher triglyceride levels but normal cholesterol levels? This study is testing whether a protein supplement called carnitine can help prevent blood vessel stiffness in teenagers who have metabolic syndrome or other risk factors for heart and blood vessel diseases. Participants in this study must be between 13 and 19 years old and have moderate to high blood triglyceride levels but normal blood cholesterol levels. This study takes place in Houston, Texas.

Are you an adult who has overweight or obesity and would be interested in a weight-loss study? This study is testing whether a medicine called phentermine is safe and effective for treating obesity over a long period. This study has multiple locations in the United States.