New drug trial offers hope for controlling dangerous bleeding in rare disorder

This study is testing an investigational drug called eptacog beta to see if it can safely and effectively treat and prevent serious bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Six participants with a history of severe bleeding will first use their standard treatments for 3 months, then switch to using the study drug to treat serious bleeds for 6 months. An optional final phase will test if regular infusions of the drug can help prevent future bleeding.