VPB1189 - 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

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: Virtual

Presenting Author(s)

Tammuella E. Chrisentery-Singleton, MD

Chief, Pediatric Hematology
Louisiana Center For Advanced Medicine
Slidell, Louisiana, United States

Co-Author(s)

WA

W. Alan Alexander

unknown
unknown
Louisville, Kentucky, United States
AA

Ahmad Al-Sabbagh

Vice President, Medical Affairs
LFB
Norwood, Maryland, United States
DB

DANIEL BONZO

VP and Global Head of Biometry
LFB
Frammingham, Massachusetts, United States
MC

Michael Callaghan

Associate Professor of Pediatrics, Director of The Cooley & Whitten Center for Hemoglobinopathies
Central Michigan University SOM, Children’s Hospital of Michigan
etroit, Michigan, United States
Miguel Escobar, MD

Professor of Medicine, Medical Director
University of Texas Health Science Center of Houston, McGovern Medical School, Gulf States Hemophilia and Thrombophilia Center, Houston, Texas
Houston, Texas, United States
AG

Adam Giermasz

Co-Director, Hemophilia Treatment Center
University of California Davis Medical Center
Sacramento, California, United States
MH

M.Nikki Hirsh

Director of Research Programs
American Thrombosis and Hemostasis Network (ATHN)
Rochester, New York, United States
JJ

Janna Journeycake

Pediatric Hematologist
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
SN

Sonia Nasr

President
Gloval
Broomfield, Colorado, United States
RP

Rajiv Pruthi

MBBS
Mayo Clinic
Rochester, Minnesota, United States
DQ

Doris V. Quon

Medical Director, Orthopaedic Hemophilia Treatment Center
Luskin Orthopaedic Institute for Children
Los Angeles, California, United States
AR

Amina Rafique

Adult Hematologist
Louisiana Comprehensive Hemophilia Center; Tulane University Medical Center
New Orleans, Louisiana, United States
MR

Mark T. Reding

Professor and Director
Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN, USA
Minneapolis, Minnesota, United States
SS

Spencer Sullivan

Chief Executive Officer, Hematology
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
Michael Recht, MD, PhD, MBA

Chief Science Officer
American Thrombosis and Hemostasis Network
Rochester, New York, United States

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.