Clinical Trials

Do you or your child have Williams-Beuren syndrome (WS) or Supravalvular Aortic Stenosis (SVAS)? Symptoms of both conditions include vascular problems that can be mild or serious. Researchers want to find out why only some people with WS and SVAS have serious symptoms while others do not. Participants in this study will provide blood or saliva samples to help researchers see what DNA or environmental changes affect the severity of the disease. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have primary ciliary dyskinesia (PCD) or primary immune deficiency (PID)? This study aims to identify genes that cause these two conditions, which can have similar symptoms. The results from this study will help researchers find new ways to treat breathing and hearing problems caused by these two conditions in a cross-sectional cohort of patients with PCD and PID. Participants in this study must be 5 to 45 years old. This study is taking place at multiple locations in the United States and in Canada.

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.

Are you between 40 and 85 with worsening idiopathic pulmonary fibrosis? This study aims to test a new combination treatment for acute exacerbations, a life-threatening complication of idiopathic pulmonary fibrosis (IPF) that has no approved treatment. The treatment is meant to help stop part of the immune system from further injuring the lung tissue. To participate in this study, you must be between 40 and 85 years old and have worsening IPF. This study is located in Birmingham, Alabama; Boston, Massachusetts; Philadelphia, Pennsylvania; and Pittsburgh, Pennsylvania.

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.

Are you between 14 and 22 weeks pregnant, and think that you might have obstructive sleep apnea (OSA) ? This study is testing whether using CPAP to treat obstructive sleep apnea in pregnant women can help lower the risk of hypertensive disorders during pregnancy. To participate in this study, you must be at least 18 years old. This study takes place in multiple locations in the United States.