Clinical Trials

Do you or your child have sickle cell disease and receive blood transfusion treatment? This study aims to determine how long red blood cells survive in people with sickle cell disease. Some people receive blood transfusions from donor blood as a treatment for sickle cell disease. But sometimes, donor blood cells do not survive long once transfused, which can make long-term blood transfusions less effective as a treatment option. Participants in this study must be 6 years old or older and have already been receiving blood transfusion treatment for more than 3 months. This study takes place in Atlanta, Georgia.

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.

Are you a woman 18 years old or older who has been diagnosed with LAM? This study is examining whether long-term treatment with medicine called sirolimus can stop lung damage in women who have LAM. This study takes place at multiple locations in the United States.