Clinical Trials

Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.

Are you pregnant and also diagnosed with high blood pressure? This study aims to study the benefits and risks of medicine to treat pregnant women who have mild high blood pressure (

Do you or your child have sickle cell disease? This study aims to develop a mathematical equation to estimate the functioning of the kidneys in children and adults with sickle cell disease. To participate in this study, you or your child must be between 5 and 50 years old. This study takes place in Birmingham, Alabama; Chicago, Illinois; and Memphis, Tennessee.

Are you or your child between 12 and 35 years old, currently using an inhaler to treat asthma, and interested in supporting research into telehealth to control and improve asthma symptoms? This study is evaluating the use of video telehealthcare to help adolescents and young adults with asthma take their medication correctly and on time. This study takes place in multiple locations across the United States.

Have you or your child been diagnosed with cystic fibrosis, and are you interested a trial medicine already in use for some people living with the condition? A medicine called Trikafta is used to treat cystic fibrosis that is caused by a specific gene mutation. This study is testing whether Trikafta can also be used to treat cystic fibrosis that is caused by other gene mutations. Participants in this study must be at least 12 years old. This study takes place in Birmingham, Alabama; Atlanta, Georgia; and Houston, Texas.

Do you have a child 2 years or younger who has had surgery to treat myelomeningocele (MMC)? Sleep – disordered breathing (SBD) is common in infants and young children who have brain and spinal cord issues, including MMC, a serious type of spina bifida. This study is testing whether treating MMC by surgery before or after birth can help lower the risk of SBD. The study will also examine the link between SBD and brain development. This study takes place in multiple locations in the United States.

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.

Is your newborn in the neonatal intensive care unit at St. Louis Children’s Hospital? This study aims to better understand breathing problems and blood flow through the heart in premature newborns to better detect conditions such as pulmonary hypertension. To participate in this study, your newborn must have been born prematurely between 24 and 29 weeks’ gestation and be a patient in the Neonatal Intensive Care Unit at St. Louis Children’s Hospital. This study is located in St. Louis, Missouri.

Do you or your child have obesity and high blood cholesterol or triglycerides? This study is testing the use of a statin in young people to see whether early treatment of high blood cholesterol can affect the risk of developing plaque in blood vessels. Participants in this study must be age 10-17, have a body mass index (BMI) in the obese range, be fluent in English, and not be pregnant.

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.