Abstract Number: VPB1189
Meeting: ISTH 2022 Congress
Background: A new recombinant activated factor VII, eptacog beta (SEVENFACT®, rFVIIa-jncw) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of bleeding events (BEs) in individuals >12 years of age with hemophilia A or B (HAB) with inhibitors. To date, no studies designed to assess safety of treatment of breakthrough BEs in people on emicizumab with eptacog beta have been performed.
Aims: We plan to evaluate the safety of eptacog beta when used to treat BEs.
Methods: ATHN 16 (NCT04647227) is a phase IV, United States-centric, multi-center, open-label safety study enrolling participants with hemophilia A or B (HAB) with inhibitors, aged 12 to 65 years, who may need to control a BE. The maximum study duration for any participant is up to 2 years from the time of enrollment. We plan to collect data on 100 BEs.
Results: At the time of abstract submission, ATHN 16, having received central IRB approval, is being rolled out across the United States, at the American Thrombosis and Hemostasis Network (ATHN), ATHN-affiliated hemophilia treatment centers. There are 22 participating sites. Accrual has been initiated, two participants provided informed consent and were enrolled. Reporting will include BE details, and safety data for 28 to 55 participants. There are no pre-specified efficacy endpoints.
Conclusion(s): ATHN 16 will explore the safety of eptacog beta as therapy for BEs in participants with HAB complicated by inhibitors with or without concurrent prophylactic treatment. As the first interventional study sponsored by ATHN, ATHN 16 represents a crucial step forward in ATHN’s clinical research capabilities.
To cite this abstract in AMA style:Chrisentery-Singleton T, Alexander W, Al-Sabbagh A, Bonzo D, Callaghan M, Escobar M, Giermasz A, Hirsh M, Journeycake J, Nasr S, Pruthi R, Quon D, Rafique A, Reding M, Sullivan s, Recht M. ATHN 16: Safety of Coagulation Factor VIIa (recombinant)-jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia A or B with Inhibitors, with or without Prophylactic Treatment [abstract]. https://abstracts.isth.org/abstract/athn-16-safety-of-coagulation-factor-viia-recombinant-jncw-for-the-treatment-of-bleeding-events-in-patients-with-congenital-hemophilia-a-or-b-with-inhibitors-with-or-without-prophylactic-treatment/. Accessed October 1, 2023.
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