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.

DIVA is a pragmatic randomized clinical trial (RCT) to determine: among (P) preterm infants born 24-27 6/7 weeks gestation undergoing extubation from mechanical ventilation, whether (I) Non-invasive neurally adjusted ventilatory assist (NIV-NAVA) (C) compared with Non-synchronized nasal intermittent positive pressure ventilation (NS-NIPPV), will reduce the incidence of (O) extubation failure within (T) 5 days (120 hours) of extubation.

The purpose of this study is to non-invasively characterize the fibrotic consequences of single ventricle physiology, its possible solution and effect on lymphatics. This project investigates the response to acute imposition of Fontan hemodynamics by examining the interrelationship between liver and cardiac fibrosis/dysfunction and lymphatic congestion along with a pilot trial of the antifibrotic agent, spironolactone, to prevent these consequences and to determine if MRI can discern these differences. The combination of serum biomarkers and MRI form a powerful non-invasive tool in putting together this complicated web of dysfunction.

Do you or your child have Friedrich's Ataxia? This study is looking into a supplement in the form of vitamin B3 plus exercise can help the body better use oxygen for people with Friedrich’s Ataxia. You must be 10 to 40 years old to join. Participants may be asked to take the daily supplement, exercise, or do both during the study. The study is taking place in Philadelphia, Pennsylvania.