NCT04520254 (clinical trial)

NCT04520254 is a clinical trial studying how well a new treatment works for a condition called hemophilia A, where blood doesn't clot properly due to missing or defective factor VIII. The trial tests a gene therapy aimed at providing a long-lasting solution by introducing a functional gene to help the body produce factor VIII naturally. Participants receive this experimental treatment to see if it safely improves their blood clotting ability over time, potentially reducing or eliminating the need for regular factor VIII injections. The goal is to develop a one-time treatment that offers sustained bleeding control.