Clinical Trials

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.

This is an open-label study of the combination of atomoxetine and oxybutynin (ato-oxy) in children with Down syndrome and obstructive sleep apnea (OSA) documented by polysomnography (PSG).

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 severe asthma that is not easily controlled by inhaled corticosteroids? This study evaluates the use of five different asthma medicines to treat adults and adolescents with severe asthma. To participate in this study, you or your child must be 12 years old or older. This study takes place in multiple locations across the United States.

Have you or your child been diagnosed with cystic fibrosis, and would you like to support better diagnosis of lung infections? This study is exploring new ways to diagnose bacterial lung infections in people who have cystic fibrosis. The study will test whether analyzing a participant’s breath and sputum samples can help diagnose infections. Participants in this study must be at least 3 years old. This study is taking place in multiple locations in the United States.

Is your child an individual with Down syndrome and obstructive sleep apnea? This study is examining whether a combination medication therapy that includes atomoxetine and oxybutynin can help treat obstructive sleep apnea in children with Down syndrome. Participants in this study must be between 6 and 17 years old. This study takes place in Tucson, Arizona.

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.

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.