Clinical Trials

Have you or your child been diagnosed with aplastic anemia but not been treated yet? This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. Participants in this study must be 2 years or older and have severe aplastic anemia that has not yet been treated. This study is located in Bethesda, Maryland.

Do you or your child have severe aplastic anemia? This study tests the safety and effectiveness of two medicines called cyclosporine and eltrombopag in people with severe aplastic anemia. The typical treatment is a combination of three medicines: cyclosporine, eltrombopag, and horse anti-thymocyte globulin. Researchers want to see if starting people at a lower dose of cyclosporine and eltrombopag before giving them horse anti-thymocyte globulin is helpful. Participants in this study must be 4 years or older with severe aplastic anemia. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you have severe aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria and a relative who is willing to donate blood plasma? This study is exploring whether these conditions can be treated using peripheral blood hematopoietic stem cells, which are easier to collect than bone marrow cells. To participate, you must be between 4 and 55 years old with severe aplastic anemia, myelodysplastic syndrome or paroxysmal nocturnal hemoglobinuria and have a relative who is between 4 and 75 years old who can donate peripheral blood stem cells. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have severe aplastic anemia or myelodysplastic syndrome? This study aims to find new ways to make stem cell transplants safer and more effective. Researchers are testing if treating people with severe aplastic anemia with a co-infusion of blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor is safe and effective. To participate in this study, you must be between 4 and 75 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.

This study is testing whether a new imaging technique can help healthcare providers diagnose venous thromboembolism. To participate in this study, you must be at least 18 years old and diagnosed with venous thromboembolism. This study takes place in Boston, Massachusetts.

Are you a healthy adult between 40 and 90 years old and willing to help research? This study will test whether a new device called the OsciPulse system, which is designed to repeatedly compress a person’s legs, can improve blood flow through the legs to prevent or treat varicose veins. To participate in this study, you must be 40 to 90 years old and healthy. This study takes place in Philadelphia, Pennsylvania.

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.

The purpose of this trial is to evaluate if augmenting a usual audit and feedback implementation approach with telehealth-enabled support improves coordinated spontaneous awakening/breathing trials and patient outcomes for mechanically ventilated patients.

Ventilated pediatric patients are frequently over-sedated and the majority suffer from delirium, a form of acute brain dysfunction that is an independent predictor of increased risk of dying, length of stay, and costs. Universally prescribed sedative medications-the GABA-ergic benzodiazepines-worsen this brain organ dysfunction and independently prolong duration of ventilation and ICU stay, and the available alternative sedation regimen using dexmedetomidine, an alpha-2 agonist, has been shown to be superior to benzodiazepines in adults, and may mechanistically impact outcomes through positive effects on innate immunity, bacterial clearance, apoptosis, cognition and delirium. The mini-MENDS trial will compare dexmedetomidine and midazolam, and determine the best sedative medication to reduce delirium and improve duration of ventilation, and functional, psychiatric, and cognitive recovery in our most vulnerable patients-survivors of pediatric critical illness.

Is your newborn in the NICU at Holtz Children’s Hospital in Miami? Newborns born very prematurely often need oxygen therapy or ventilation to help them breathe and survive. This study will help doctors understand how changes in oxygen and carbon dioxide levels while newborns are getting treatment affect how their lungs develop. While your newborn is in the newborn neonatal intensive care unit (NICU), researchers will record his or her oxygen and carbon dioxide levels, heart rate, and other measures. To participate in this study, your newborn must have been born prematurely between 23 and 28 weeks’ gestation, be less than 28 days old, and be receiving oxygen therapy. This study is located in Miami, Florida.