Clinical Trials

Do you or your child have sickle cell disease and receive blood transfusion treatment? This study aims to determine how long red blood cells survive in people with sickle cell disease. Some people receive blood transfusions from donor blood as a treatment for sickle cell disease. But sometimes, donor blood cells do not survive long once transfused, which can make long-term blood transfusions less effective as a treatment option. Participants in this study must be 6 years old or older and have already been receiving blood transfusion treatment for more than 3 months. This study takes place in Atlanta, Georgia.

Are you an adult who receives long-term red blood cell transfusion therapy for sickle cell disease? This 3-month study tests whether donated blood that is more than 30 days old leads to more infections in people with sickle cell disease than blood that is less than 10 days old. Some hospitals around the United States have requirements regarding the age of donor blood. This research aims to inform new evidence-based policies on the age and use of donor blood in people with sickle cell disease that rely on blood transfusions. To participate in this study, you must be between 16 and 45 years old, have sickle cell disease, and be receiving long-term red blood cell transfusion therapy. This study takes place in Atlanta, Georgia; Chapel Hill, North Carolina; and Milwaukee, Wisconsin.

Are you or your child between the ages of 3 and 21 and experiencing pain episodes? This study tests a treatment called arginine therapy for pain episodes in children with sickle cell disease. Researchers are investigating how safe and effective this treatment is, as well as how arginine is metabolized in the body and its effects on cells. To participate in this study, you or your child must have sickle cell disease and pain that requires medical attention. This study takes place in multiple locations in the United States.

Have you or your child been diagnosed with cystic fibrosis, and are you interested a trial medicine already in use for some people living with the condition? A medicine called Trikafta is used to treat cystic fibrosis that is caused by a specific gene mutation. This study is testing whether Trikafta can also be used to treat cystic fibrosis that is caused by other gene mutations. Participants in this study must be at least 12 years old. This study takes place in Birmingham, Alabama; Atlanta, Georgia; and Houston, Texas.

Are you a woman 18 years old or older who has been diagnosed with LAM? This study is examining whether long-term treatment with medicine called sirolimus can stop lung damage in women who have LAM. This study takes place at multiple locations in the United States.

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 study is to investigate that the worsening orthostatic tachycardia and symptoms after glucose ingestion in POTS patients are due to a greater increase in splanchnic venous capacitance and excessive blood pooling during an orthostatic challenge.

Do you have a family history of high cholesterol? Researchers are studying whether a particular medication can lower cholesterol in people with a family history of high cholesterol. You may be able to participate if you are 18 to 69 years old, have not had a heart attack or stroke recently, and do not have uncontrolled high blood pressure. This study will take place in Nashville, Tennessee.

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 purpose of this study is to compare reintubation rates and outcomes of patients treated with high-flow nasal canula oxygen therapy (HFNC) and patients treated with provider choice of standard care.