Clinical Trials

Are you or your child at least 13 years old and have sickle cell disease? In this stidy, researchers are trying to find out if a new medication improves the success of a blood stem cell transplant. People 13 years old or older who are eligible for a blood stem cell transplant to treat sickle cell disease may be able to participate. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood for the study. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

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.

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.