Evaluating Eptacog Beta for Treating and Preventing Bleeding in People with Glanzmann Thrombasthenia

What this Trial Is About

Researchers are evaluating eptacog beta EB, an investigational drug not yet approved by the FDA, for treating and preventing acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. This Phase 2 study includes participants with severe bleeding symptoms and aims to assess EBs effectiveness and safety in managing serious bleeding events and preventing future bleeds during an optional extension phase. The study begins with a 3-month non-interventional phase where participants use their standard treatments for bleeding events while recording these episodes. Afterward, for 6 months, participants will use eptacog beta on-demand to treat breakthrough bleeding, with doses given intravenously at 75 mcgkg every 3 hours as needed. Participants may then opt into a 6-month extension phase where EB is given routinely 2 to 3 times per week to prevent bleeding complications. Participants will visit their hematologist about 4 to 5 times over the 9-month study and keep a diary logging bleeding events and treatments. They will complete questionnaires on how bleeding affects their daily life. Blood samples will be collected to monitor their condition and potential side effects. The study will evaluate how effectively EB controls bleeding within 24 hours and compare bleeding rates between the on-demand and prophylaxis phases.

Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)