SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Safety of SEVENFACT® for the Treatment of Bleeding Events in Patients With Hemophilia A or B With Inhibitors
1 other identifier
interventional
55
1 country
21
Brief Summary
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on prophylactic treatment who may need to control a BE.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started Jun 2021
Longer than P75 for phase_4
21 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 23, 2020
CompletedFirst Posted
Study publicly available on registry
November 30, 2020
CompletedStudy Start
First participant enrolled
June 28, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2027
January 12, 2026
January 1, 2026
5.3 years
November 23, 2020
January 9, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of participants and percentage of Safety Events (AEs)
Adverse Events and SAEs defined by the European Haemophilia Safety Surveillance System 2018 (EUHASS) and Serious Adverse Events (SAEs) as defined by the US Food and Drug Association.
From time of consent through BE onset until 3 days after last dose of SEVENFACT®.
Study Arms (1)
Hemophilia A and B Cases
OTHERSEVENFACT® has been approved for the treatment of bleeding events in individuals with hemophilia A or B with inhibitors. This study is intended to further investigate the safety and tolerability of SEVENFACT in participants with hemophilia A or B with inhibitors in the presence or absence of prophylactic therapies. Dosing will be at the discretion of the attending physician, and each participant will be supplied with the equivalent of nine 75 µg/kg doses, which aligns with the recommended dosing schedule as provided in SEVENFACT's United States Prescribing Information (USPI). In the event of a bleeding episode (BE), the participant will either self-administer the correct dose under the guidance of the treating investigator or the dose will be administered at a treatment facility.
Interventions
a transgenically produced, activated, recombinant, human factor VII (rhFVIIa) protein with the brand name of SEVENFACT®. This protein is a clotting factor in the coagulation cascade that is produced in and purified from the milk of transgenic rabbits. SEVENFACT is approved for the treatment and control of bleeding episodes occurring in adults and adolescents (12 years of age and older) with hemophilia A or B with inhibitors.
Eligibility Criteria
You may qualify if:
- Have a diagnosis of hemophilia A or B with inhibitors.
- Be 12 years of age and older
- Be capable of understanding and willing to comply with the conditions of the protocol or have a legal guardian who is capable of understanding and complying with the conditions of the protocol
- Have read, understood, and documented written informed consent/assent
- Be able to provide medical evidence through prior medical history of previous inhibitor levels
- Be willing and able to use the ATHN mobile application or a paper diary to document BEs and medication usage
You may not qualify if:
- Have a disorder of hemostasis in addition to Hemophilia A or B
- Have a known or suspected intolerance or hypersensitivity to SEVENFACT® or its ingredients
- Have a known allergy or hypersensitivity to rabbits or rabbit proteins
- Are receiving prophylactic treatment for bleeding with a drug or biologic that is not approved for this use by the FDA
- Have had implantation of an investigational medical device within the prior 6 months
- Have received an investigational drug within 30 days of the baseline visit
- Have an elective surgical procedure planned during the duration of their participation in the study\*
- Have any life-threatening disease, or other disease or condition which, in the investigator's judgment, could pose a potential hazard to the patient or interfere with study participation or study outcome (e.g., a history of non responsiveness to bypassing products or thromboembolic disease)
- Should a participant require an unplanned surgery, the participant will not be withdrawn from the study unless the investigator deems it necessary. Instead, the participant will receive standard of care treatment as determined by the attending physician. If the participant is not withdrawn from the study, the participant's participation in the study will be paused until the investigator feels it is safe for them to continue.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- LFB USA, Inc.collaborator
- American Thrombosis and Hemostasis Networklead
Study Sites (21)
Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Arkansas Center for Bleeding Disorders
Little Rock, Arkansas, 72202, United States
Orthopaedic Institute for Children
Los Angeles, California, 90007, United States
University of California at Davis UC Davis Hemostasis and Thrombosis Center
Sacramento, California, 95817, United States
Children's National Hemophilia Center
Washington D.C., District of Columbia, 20010, United States
Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders
Orlando, Florida, 32806, United States
Hemophilia of Georgia Center for Bleeding and Clotting Disorders of Emory, Adult Division
Atlanta, Georgia, 31404, United States
Willett Children's Hospital at Memorial University Medical Center
Savannah, Georgia, 31404, United States
Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
Boston, Massachusetts, 02114, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Henry Ford Health System
Detroit, Michigan, 48202, United States
MSU Center for Bleeding and Clotting Disorders
Lansing, Michigan, 48912, United States
Center for Bleeding and Clotting Disorders, University of Minnesota
Minneapolis, Minnesota, 55455, United States
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905, United States
Kansas City Regional Hemophilia Center
Kansas City, Missouri, 64108, United States
Northwell Health, Long Island Jewish
New Hyde Park, New York, 11040, United States
Brody School of Medicine at East Carolina University
Greenville, North Carolina, 27834, United States
University Hospitals Health System Cleveland
Cleveland, Ohio, 44106, United States
Oklahoma Center for Bleeding and Clotting Disorders
Oklahoma City, Oklahoma, 73104, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
Gulf States Hemophilia and Thrombophilia Center-University of Texas Health Science Center @Houston
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Tammuella Chrisentery-Singleton, MD
American Thrombosis and Hemostasis Network
- PRINCIPAL INVESTIGATOR
Mark Reding, MD
University of Minnesota
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 23, 2020
First Posted
November 30, 2020
Study Start
June 28, 2021
Primary Completion (Estimated)
September 30, 2026
Study Completion (Estimated)
March 31, 2027
Last Updated
January 12, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share