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.

The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.

Do you or your child have sickle cell disease and also have an interest in contributing to long-term data collection? This study is monitoring patients and collecting biological samples from patients who have sickle cell disease to better understand long-term outcomes. Participants can be any age and must have sickle cell disease. This study is located in six sites in Illinois, Louisiana, North Carolina, and Tennessee.

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 or a family member have pulmonary hypertension? This study is looking at how hormones contribute to pulmonary arterial hypertension and whether treatments for hormone imbalances or insulin resistance also improve pulmonary hypertension. To participate in this study, you must be 90 years old or younger and have pulmonary arterial hypertension or be a family member of someone who has this condition. This study is located in Nashville, Tennessee.

Do you or your child have sickle cell disease? This study aims to develop a mathematical equation to estimate the functioning of the kidneys in children and adults with sickle cell disease. To participate in this study, you or your child must be between 5 and 50 years old. This study takes place in Birmingham, Alabama; Chicago, Illinois; and Memphis, Tennessee.