Clinical Trials

This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are currently transfused with D- RBC (Red Blood Cell) units.

This study examines cardiovascular responses to a brief psychological challenge under laboratory conditions among adult volunteers. The study is designed to examine associations between transient changes in cardiovascular activity induced by the psychological task, measures of overall cardiovascular health, and measures of cerebrovascular health measured by magnetic resonance imaging. Study results are expected to provide new information about the relationships between cardiovascular activity changes to psychological challenges and cardiovascular and cerebrovascular health in adults.

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.

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.

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.

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.

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.

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.

Do you or a loved one have pneumonia? This study will test whether combining two medicines to reduce inflammation of the airway and to keep it open may help prevent respiratory failure in people who have pneumonia. To participate in this study, you must be at least 18 years old and have severe pneumonia with low blood oxygen levels.

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.