Clinical Trials

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 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 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.

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.

Are you or your child between the ages of 3 and 21 and experiencing pain episodes? This study tests a treatment called arginine therapy for pain episodes in children with sickle cell disease. Researchers are investigating how safe and effective this treatment is, as well as how arginine is metabolized in the body and its effects on cells. To participate in this study, you or your child must have sickle cell disease and pain that requires medical attention. This study takes place in multiple locations in the United States.

Does your child have overweight or obesity? This study is exploring the link between obesity and sleep deprivation in children. Researchers will test whether participants who do not get enough good quality sleep have trouble making healthy food choices. Participants in this study must be 8 to 12 years old and have overweight or obesity. This study takes place in Pittsburgh, Pennsylvania; and Providence, Rhode Island.

Are you an adult with obesity and at high risk of heart and blood vessel disease? This study is testing a new program to help people who have obesity stick to their healthy eating plan. Researchers from this study are developing a program that will predict when participants are most likely to stop making healthy food choices and will send messages to remind participants of their weight loss goals. To participate in this study, you must be 18 to 70 years old, have overweight or obesity, and be at high risk of heart and blood vessel diseases. This study takes place in Providence, Rhode Island.

Does your child have overweight or obesity or just want to be in a study on sleep and eating habits? Overweight and obesity are common in children who do not get enough good quality sleep. This study aims to understand how sleep deficiency can affect a child’s ability to make good food choices. Researchers will ask participants to spend either 8 or 11 hours in bed and to keep track of their food choices. Participants in this study must be 8 to 12 years old. This study takes place in Pittsburgh, Pennsylvania and Providence, Rhode Island.