The purpose of this study is to learn more about using the study drug, CTX001, to treat sickle cell disease in children. We want to see how effective it is and if there are any side effects.
Study Topics
Sickle Cell Disease
Children, Infants, Teen Health
The purpose of this study is to learn more about a new medicine called etavopivat. Researchers want to see if it can help lower sickle cell pain crises and improve energy, blood health, and daily life. A sickle cell pain crisis is a sudden time of strong pain that happens when sickle‑shaped blood cells block blood flow in small blood vessels. This study is for volunteers who have had 2 to 15 sickle cell pain crises in the past year.
Study Topics
Sickle Cell Disease
