Clinical Trials

The goal of this work is to develop and test a noninvasive ultrasound-based imaging technology to better identify patients at high risk of stroke so that appropriate and timely intervention may be administered to prevent it.

Do you have post-traumatic stress disorder (PTSD) or have experienced a psychosocial trauma, or want to help research? This study uses positron emission tomography/magnetic resonance imaging (PET/MRI) to look at the relationship between psychosocial stress and systemic inflammation/atherosclerosis. This study is located in Boston, Massachusetts, and New York, New York.

Do you have symptoms of a lung disease (such as long-term wet cough, bronchiectasis, or recurrent pneumonia) with no genetic diagnosis? This study aims to develop new ways to diagnose primary ciliary dyskinesia (PCD) or primary immune deficiency (PID), two conditions that can have similar symptoms. Researchers will combine information from genetic testing, lung imaging, and lung function tests to diagnose and tell the difference between these two conditions. Participants in this study must be 5 to 45 years old and must have symptoms of a serious lung disease but no diagnosis. This study is taking place at multiple locations in the United States and in Canada, including the NIH Clinical Center in Bethesda, Maryland.

Do you or your child have PCD and want to help researchers better understand how cilia are formed? This study aims to identify the genes that control how cilia are formed. Cilia are hairlike structures that line the airways and protects the normal lung. The study will also identify how mutations to these genes can cause PCD. This study takes place in Chapel Hill, North Carolina.

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.

Do you or your child have symptoms of a serious lung disease but no diagnosis? This study aims to develop new ways to diagnose primary ciliary dyskinesia (PCD) and primary immune deficiency (PID), two conditions that can have similar symptoms. Researchers will combine information from genetic testing, lung imaging, and lung function tests to diagnose ant tell the difference between these two conditions. 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.

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.

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.