Clinical Trials

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.

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.

Do you want to participate in a study with your child who has cystic fibrosis? This study is exploring computer models to compare cell samples from the noses of people who have cystic fibrosis, their parents, and other healthy individuals in hopes of better predicting how patients will respond to medicine. These computer models may allow medicines to be developed more rapidly and better predict who will respond to a certain cystic fibrosis treatment. To participate in this study, you must fall into one of the following groups: (1) have cystic fibrosis and be at least 12 years old, (2) be the biological parent of a cystic fibrosis patient enrolled in the study and be at least 18 years old, or (3) have no history of lung disease and be at least 18 years old. This study is located in Pittsburgh, Pennsylvania.

This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are currently transfused with D- RBC (Red Blood Cell) units.

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.

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.

Do you or your child have sickle cell disease and also have an interest in contributing to long-term data collection? This study is monitoring patients and collecting biological samples from patients who have sickle cell disease to better understand long-term outcomes. Participants can be any age and must have sickle cell disease. This study is located in six sites in Illinois, Louisiana, North Carolina, and Tennessee.

Are you an adult with sickle cell disease? This study aims to determine whether monthly exchange blood transfusions (removing blood and replacing it with transfused red blood cells) can improve health and prevent or reduce the number of pain crises. To participate in this study, you must be at least 18 years old, have sickle cell disease, and have not been on a chronic exchange transfusion program for at least 2 months. This study is located in Pittsburgh, Pennsylvania.

Do you have sickle cell disease and need chronic red blood cell (RBC) transfusions? This study aims to determine the effectiveness of matching donor red cells by RH genotype for people who need frequent RBC transfusions. Participants in this study must be 1 year or older. This study is located in Philadelphia, Pennsylvania.

Are you an adult with sickle cell disease who’s recently had a vaso-occlusive episode? This study investigates better ways for emergency departments (EDs) to manage vaso-occlusive episodes, or pain crises, in people with sickle cell disease. This study is incorporating “Individualized Pain Plans” into patients’ electronic health records to allow for faster and more accurate treatment in the ED. To participate in this study, you must be between the ages of 18 and 45 and have sickle cell disease. You must also have had at least one visit to a participating ED in the past 90 days due to pain from a vaso-occlusive episode and at least one visit at the study site sickle cell disease clinic within the past 12 months. This study takes place in multiple locations in the United States.