Clinical Trials

his trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice.

Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor SCD and its treatment through their impact on cerebral hemodynamics, as the brain is one of the most vulnerable and consequential targets of the disease. Specifically, this study will optimize quantitative magnetic resonance imaging (MRI) and advanced optical spectroscopy techniques such as frequency-domain near-infrared and diffuse correlation spectroscopies (FDNIRS-DCS) to monitor 1) cerebral oxygen transport with measures of cerebral blood flow (CBF), cerebral oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen consumption (CMRO2) and 2) endothelial function with cerebrovascular reactivity (CVR).

The main goal of this study is to determine whether the treatment will lead to a complete absence of severe vaso-occlusive events (VOEs) in patients with severe SCD.

Do you have heart failure and live near Massachusetts General Hospital in Boston, Massachusetts? Researchers are testing whether an online program helps lower the number of times patients with heart failure return to the hospital. You may be able to participate if you are at least 18 years old, have heart failure, and live within 30 miles of Massachusetts General Hospital in Boston.

Do you have heart failure? In this clinical trial, researchers are using images of the heart and blood tests to learn more about Black and Hispanic patients with heart failure. You may be able to participate if you are at least 60 years old; of Black, Hispanic, or Caribbean origin; and have been diagnosed with heart failure. The study is taking place in locations in Connecticut, Massachusetts, and New York.

Are you an adult with heart failure? Unplanned weight loss is quite common among people with heart failure. Researchers in this clinical trial are studying whether adding a protein supplement to daily meals can help prevent muscle and weight loss for people with heart failure. To participate, you must be at least 18 years old and have heart failure. The study is taking place in Boston, Massachusetts.

Do you have mitral valve disease? This clinical trial is testing different types of images taken of the heart to learn more about mitral valve disease and the best ways to treat it. You must be 60 years old to participate. The study is enrolling patients in locations in Arkansas, California, Georgia, Indiana, Louisiana, Maine, Maryland, Massachusetts, Michigan, Missouri, New York, North Carolina, Ohio, Pennsylvania, and Quebec City, Canada.

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 open-heart surgery for your aortic valve? This study aims to look at changes in inflammation and gene activity while surgery patients are on a heart-lung bypass machine. To participate in this study, you must be at least 20 years old and be scheduled for aortic valve surgery, either with or without coronary artery bypass grafting (CABG). This study is located in Boston, Massachusetts.

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.