Clinical Trials

Have you or your child been diagnosed with aplastic anemia but not been treated yet? This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. Participants in this study must be 2 years or older and have severe aplastic anemia that has not yet been treated. This study is located in Bethesda, Maryland.

Do you or your child have severe aplastic anemia? This study tests the safety and effectiveness of two medicines called cyclosporine and eltrombopag in people with severe aplastic anemia. The typical treatment is a combination of three medicines: cyclosporine, eltrombopag, and horse anti-thymocyte globulin. Researchers want to see if starting people at a lower dose of cyclosporine and eltrombopag before giving them horse anti-thymocyte globulin is helpful. Participants in this study must be 4 years or older with severe aplastic anemia. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you have severe aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria and a relative who is willing to donate blood plasma? This study is exploring whether these conditions can be treated using peripheral blood hematopoietic stem cells, which are easier to collect than bone marrow cells. To participate, you must be between 4 and 55 years old with severe aplastic anemia, myelodysplastic syndrome or paroxysmal nocturnal hemoglobinuria and have a relative who is between 4 and 75 years old who can donate peripheral blood stem cells. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have severe aplastic anemia or myelodysplastic syndrome? This study aims to find new ways to make stem cell transplants safer and more effective. Researchers are testing if treating people with severe aplastic anemia with a co-infusion of blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor is safe and effective. To participate in this study, you must be between 4 and 75 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.

The purpose of this study is to determine the occurrence of scarring of the heart (cardiac fibrosis) and inflammation in those with perinatally acquired Human Immunodeficiency Virus (HIV) infection compared to people not infected with HIV. The information learned from this research may help the investigator to better understand the link between cardiac fibrosis and cardiac dysfunction and inflammation in those with perinatally acquired HIV infection compared to the uninfected.

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.

The purpose of this study is to see if adding a drug called Regadenoson to the EVLP circulation reservoir during perfusion of marginal donor lungs will help increase the likelihood that the donor lungs will become usable for transplantation.

The purpose of this study is to determine if acquired (partial) Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) deficiency contributes substantially to the pathogenic mechanisms underlying Chronic Rhinosinusitis (CRS), creating a localized environment that impairs mucociliary clearance (MCC).

This project will determine the health impact of parenthood on people with cystic fibrosis (CF). The study team will use retrospective data to provide relatively immediate evidence on parenthood's effect on pulmonary health.

The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhaled contrast gas, and compare these measures to lung function assessed by spirometry and multiple breath nitrogen washout. This study also looks at how these measures change in response to a pulmonary exacerbation and treatment (if applicable). Over the span of a year, participants would be asked to complete 3-5 visits to the University of North Carolina at Chapel Hill (UNC). with each lasting up to 4 hours. If participants do not have a pulmonary exacerbation during the year they would be asked to complete 3 visits (one at enrollment, a second roughly 2 weeks later, and the third approximately a year later). If participants do experience a CF pulmonary exacerbation they would complete 5 visits (Visit 1, Visit 2, two exacerbation visits with one before treatment and the other after, and Visit 3 at one year after Visit 1). Only one exacerbation per participant will be tracked. Participants are eligible for this study if they are 18 years old or older, have Cystic Fibrosis (CF) with mild lung disease (FEV1 >/= 60%), and can undergo an MRI. There are no known benefits for participating in this study.