Clinical Trials

Does your child have obesity? In this study, children and teens, 7 to 13 years of age with obesity and their parents will learn about nutrition, behavior changes, and exercise through the Bright Bodies lifestyle program. Researchers want to see if this program can help lower weight gain and screen time, improve sleep, eating behaviors, and quality of life, and reduce parental stress. The study is taking place at locations in Alabama, California, and Maine.

This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat Severe Aplastic Anemia.

The goal of this clinical trial is to compare different types of blood transfusions for critically injured patients who require large-volume blood transfusions. Participants must be at least 15 years old and need a massive blood transfusion. The study is taking place in locations in Alabama, California, Louisiana, Maryland, Missouri, North Carolina, Ohio, Oregon, Pennsylvania, Texas, Washington, and Wisconsin.

The Achieve Trial is a randomized clinical trial to test whether lowering blood pressure to less than 140/90 mmHg in women with hypertensive disorders of pregnancy will prolong pregnancy.

Have you or your child been diagnosed with a blood disorder? If so, you (or they) may be able to participate in a clinical study where researchers are studying a type of infusion using cord blood. Participants can be of any age but must have a blood disorder diagnosed by their doctor. The study is taking place in locations in Alabama, Arizona, California, Colorado, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Maryland, Massachusetts, Michigan, Minnesota, Mississippi, Missouri, Nebraska, New York, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Virginia, Washington, Washington, D.C., West Virginia, and Wisconsin.

Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.

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.

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.

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.

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.