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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

1Mississippi Center For Advanced Medicine, Slidell, Louisiana, United States, 2unknown, Louisville, Kentucky, United States, 3LFB, Norwood, Maryland, United States, 4LFB, Norwood, Massachusetts, United States, 5Central Michigan University SOM, Children’s Hospital of Michigan, etroit, Michigan, United States, 6University of Texas Health Science Center, Houston, Texas, United States, 7University of California Davis Medical Center, Sacramento, California, United States, 8American Thrombosis and Hemostasis Network (ATHN), Rochester, New York, United States, 9University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States, 10Gloval, Broomfield, Colorado, United States, 11Mayo Clinic, Rochester, Minnesota, United States, 12ORTHOPAEDIC HEMOPHILIA TREATMENT CENTER, Los Angeles, California, United States, 13Louisiana Comprehensive Hemophilia Center; Tulane University Medical Center, New Orleans, Louisiana, United States, 14University of Minnesota, Minneapolis, Minnesota, United States, 15Mississippi Center For Advanced Medicine, Madison, Mississippi, United States, 16American Thrombosis and Hemostasis Network, Rochester, New York, United States

Abstract Number: VPB1189

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Management of Bleeding and Trauma

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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ISTH Congress Abstracts - 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/