Clinical Trials

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.

Is your child having otolaryngologic surgery with anesthesia? Children with Down syndrome often experience dangerously low heart rates when they receive anesthesia for procedures. This study will look at children who have Down syndrome and children who do not to monitor their heart rate during this type of procedure. Participants in this study must be between 1 and 8 years old. This study is located in Cincinnati, Ohio.

Are you the parent or caregiver of a 12- to 15-year-old child with asthma who uses an inhaled corticosteroid to treat symptoms? This study is testing whether a mobile health tool, called AIM2ACT, can help adolescents and their caregivers manage symptoms of asthma. To participate in this study, you must be between 18 and 70 years old. This study takes place in Gainesville, Florida.

Do you or your child have severe asthma that is not easily controlled by inhaled corticosteroids? This study evaluates the use of five different asthma medicines to treat adults and adolescents with severe asthma. To participate in this study, you or your child must be 12 years old or older. This study takes place in multiple locations across the United States.

Have you or your child been diagnosed with cystic fibrosis, and would you like to support better diagnosis of lung infections? This study is exploring new ways to diagnose bacterial lung infections in people who have cystic fibrosis. The study will test whether analyzing a participant’s breath and sputum samples can help diagnose infections. Participants in this study must be at least 3 years old. This study is taking place in multiple locations in 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 or your child have primary ciliary dyskinesia (PCD)? This study aims to find out whether mild, moderate, or serious types of PCD are caused by different gene mutations. Researchers will test how well people who have various mutations, or gene changes, that cause PCD can clear mucus from their airways. Results from this study will help improve the diagnosis and treatment of PCD. This study takes place in Chapel Hill, North Carolina.

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.