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

This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition. The study will assess the effectiveness and safety of eptacog beta (EB) when used to treat serious bleeding events, and in an optional phase, when used routinely to prevent bleeding. During the first three (3) months, participants will manage any bleeding episodes with their standard treatment (e.g., factor products or platelet transfusions). After this initial period, they will use the study drug to treat serious bleeding events. Participants will have approximately 4 to 5 visits with their hematologist over the 9-month study period. They will also be asked to complete a diary documenting bleeding episodes and treatments, and to answer questions about how bleeding affects their daily life. Blood samples will be collected to monitor their condition and any potential side effects of the study drug. At the end of the main study, participants will have the option to enter an optional extension phase, where they will receive routine intravenous infusions of the study drug 2 to 3 times per week for 6 months to help prevent future bleeding episodes and complications.

This study aims to investigate eptacog beta (EB), a new form of recombinant activated factor VIIa, for bleeding management in persons with Glanzmann thrombasthenia regardless of platelet refractoriness status. Investigators will enroll six (6) people (adult or pediatric) with Glanzmann thrombasthenia (GT) who have a severe bleeding phenotype (defined as ≥ 2 treated bleeding events in the past 12 months, ≥ 1 hospitalization for bleeding or severe anemia or requiring prophylactic therapy to prevent bleeding). Initially, a retrospective chart review will collect data regarding bleeding events and their management over the previous 6 months before enrollment. The initial 3 months of this trial will be a non-interventional phase during which time, participants will receive standard-of-care on-demand therapy for acute bleeding events at the discretion of their hematologist. They will complete a diary logging their bleeding events and their management. Hemostatic efficacy following acute bleed treatment will be evaluated using a 4-point hemostasis efficacy score. Following this initial non-interventional phase, participants will use eptacog beta (EB) 75 mcg/kg/dose every 3 Hours for the management of breakthrough bleeding episodes that are not responsive to local hemostatic control and anti-fibrinolytic therapies, with the precise frequency of infusions and duration of treatment at the discretion of the subject's treating physician. This on-demand interventional phase will last 6 months. During this time, participants will log their acute bleeding episodes and management. At varying time points following a dose of EB, participants will complete a 4-point hemostasis scale assessing the efficacy of EB in controlling bleeding. Following the 6-month interventional on-demand phase, participants may choose to continue in an interventional prophylaxis arm.