Clinical Trials

Are you 15 to 40 years old and have severe sickle cell disease? This study is comparing long-term outcomes for patients who receive blood and bone marrow transplants and those who receive standard treatment with medicines. Participants also have an option of contributing blood samples to be stored for future research. Participants must be 15 to 40 years old and have severe sickle cell disease. This study is located in Madison, 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.

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 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.

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.

Does your child have Down Syndrome? Did they have a complete atrioventricular septal defect (CAVSD) repair within the first year of their life? Researchers in this study are investigating how the
brain develops in children with Down Syndrome who have congenital heart disease. To participate, your child must be between 5 and 12 years old and have Down Syndrome. The study will enroll children who have congenital heart disease as well as children who do not have congenital heart disease. The study is taking place in locations in Delaware, Georgia, Indiana, Massachusetts, Michigan, Missouri, New York, Ohio, Pennsylvania, South Carolina, Texas, Utah, Washington, D.C., and Toronto, Canada

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.

Do you or one of your children have a congenital heart defect? This study will find both common genetic causes of congenital heart disease and ways that genes influence results of medical treatment. To participate in this study, you or your child must have congenital heart disease. This study is located in Los Angeles, Palo Alto, and San Francisco, California; New Haven, Connecticut; Boston, Massachusetts; New York and Rochester, New York; Philadelphia, Pennsylvania; and Salt Lake City, Utah.

Does your infant have a low platelet count? This study investigates immature platelet counts as a marker for bleeding risk in newborns with thrombocytopenia, compared with platelet counts alone. Immature platelets are the most recently produced platelets and may be a better marker of platelet production. To participate in this study, you must have a newborn who is less than 32 weeks’ gestational age, has a birth weight greater than 500 grams, and has a platelet count less than 100 × 109/L. This study takes place in multiple locations in the United States, the Netherlands, and Sweden.

Are you an adult with polycythemia vera or thrombocytosis? This study aims to find a new treatment or cure for polycythemia vera and thrombocytosis by locating genes and their changes, or mutations, that may contribute to these disorders. To participate in this study, you must have polycythemia vera with elevated hemoglobin (higher than 18 in men and 16 in women) or thrombocytosis with an elevated platelet count (higher than 450,000). This study takes place in Salt Lake City, Utah.