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 or one of your children have a congenital heart defect? This study will find both common genetic causes of congenital heart disease and ways that genes influence results of medical treatment. To participate in this study, you or your child must have congenital heart disease. This study is located in Los Angeles, Palo Alto, and San Francisco, California; New Haven, Connecticut; Boston, Massachusetts; New York and Rochester, New York; Philadelphia, Pennsylvania; and Salt Lake City, Utah.

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 your child have pulmonary hypertension and want to help research? This study is looking to better understand pulmonary hypertension in children who have this condition. To participate in this study, you or your child must be 21 years old or younger and must have been diagnosed with pulmonary hypertension before the age of 18. This study is located at multiple sites across the United States and Canada, including California, Colorado, Massachusetts, New York, Pennsylvania, Tennessee, and Alberta, Canada.

Is your newborn in the neonatal intensive care unit at St. Louis Children’s Hospital? This study aims to better understand breathing problems and blood flow through the heart in premature newborns to better detect conditions such as pulmonary hypertension. To participate in this study, your newborn must have been born prematurely between 24 and 29 weeks’ gestation and be a patient in the Neonatal Intensive Care Unit at St. Louis Children’s Hospital. This study is located in St. Louis, Missouri.

Do you want to participate in a study with your child who has cystic fibrosis? This study is exploring computer models to compare cell samples from the noses of people who have cystic fibrosis, their parents, and other healthy individuals in hopes of better predicting how patients will respond to medicine. These computer models may allow medicines to be developed more rapidly and better predict who will respond to a certain cystic fibrosis treatment. To participate in this study, you must fall into one of the following groups: (1) have cystic fibrosis and be at least 12 years old, (2) be the biological parent of a cystic fibrosis patient enrolled in the study and be at least 18 years old, or (3) have no history of lung disease and be at least 18 years old. This study is located in Pittsburgh, Pennsylvania.

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.

Is your child black and 8 to 11 years old? This study is testing a way of enhancing sleep in children with the goal of preventing obesity. To participate in this study, your child must be 8 to 11 years old and must be black or African American. This study is located in Philadelphia, Pennsylvania.

Do you have symptoms of a lung disease (such as long-term wet cough, bronchiectasis, or recurrent pneumonia) with no genetic diagnosis? This study aims to develop new ways to diagnose primary ciliary dyskinesia (PCD) or primary immune deficiency (PID), two conditions that can have similar symptoms. Researchers will combine information from genetic testing, lung imaging, and lung function tests to diagnose and tell the difference between these two conditions. Participants in this study must be 5 to 45 years old and must have symptoms of a serious lung disease but no diagnosis. This study is taking place at multiple locations in the United States and in Canada, including the NIH Clinical Center in Bethesda, Maryland.