Clinical Trials

The investigators propose to study the relationship between right ventricle (RV) steatosis and RV function, exercise capacity, and outcomes in humans with pulmonary arterial hypertension (PAH) and to identify potential drivers of lipid accumulation.

The Investigators propose a randomized trial of smart texts versus usual care for 6 months. The Investigators will randomize 100 PAH patients to the mHealth intervention or usual care. The Investigators will test the effect of a text-based mHealth intervention on HRQOL in PAH using the PAH-specific emPHasis-10 questionnaire. The Investigators will also test the effect of an mHealth intervention on exercise capacity, measured by a supervised home-based 6MWD test. Finally, the Investigators will examine the effect of the intervention on time to clinical worsening (composite of PAH therapy escalation, PAH hospitalization, and death) one year after randomization.

The investigators propose to study the relationship between right ventricle (RV) steatosis and RV function, exercise capacity, and outcomes in humans with pulmonary arterial hypertension (PAH) and to identify potential drivers of lipid accumulation.

Patients with pulmonary arterial hypertension (PAH) have reduced health related quality of life (HRQOL) and impaired exercise capacity. Despite fourteen approved therapies, most patients die within ten years. Increasing physical activity is highly efficacious in PAH, resulting in six-minute walk distance (6MWD) and HRQOL improvement that often exceeds the effect of medications. Prior activity studies required inpatient rehabilitation, which is impractical, hard to sustain, and poorly scalable to a rare disease.

The Investigators propose a randomized trial of smart texts versus usual care for 6 months. The Investigators will randomize 100 PAH patients to the mHealth intervention or usual care. The Investigators will test the effect of a text-based mHealth intervention on HRQOL in PAH using the PAH-specific emPHasis-10 questionnaire. The Investigators will also test the effect of an mHealth intervention on exercise capacity, measured by a supervised home-based 6MWD test. Finally, the Investigators will examine the effect of the intervention on time to clinical worsening (composite of PAH therapy escalation, PAH hospitalization, and death) one year after randomization.

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.

Are you an adult with sickle cell disease who’s recently had a vaso-occlusive episode? This study investigates better ways for emergency departments (EDs) to manage vaso-occlusive episodes, or pain crises, in people with sickle cell disease. This study is incorporating “Individualized Pain Plans” into patients’ electronic health records to allow for faster and more accurate treatment in the ED. To participate in this study, you must be between the ages of 18 and 45 and have sickle cell disease. You must also have had at least one visit to a participating ED in the past 90 days due to pain from a vaso-occlusive episode and at least one visit at the study site sickle cell disease clinic within the past 12 months. This study takes place in multiple locations in the United States.