SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

November 20, 2023 updated by: American Thrombosis and Hemostasis Network

Safety of SEVENFACT® for the Treatment of Bleeding Events in Patients With Hemophilia A or B With Inhibitors

Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors12 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Primary Objective:

To evaluate the safety of SEVENFACT® when used to treat bleeding episodes in participants with Hemophilia A or B with inhibitors either with or without prophylactic treatment

Study Design:

Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A and B with inhibitors12 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.

Study Duration:

Participants will be followed longitudinally from the time of enrollment to the end of their participation in the study. The maximal study duration for any participant in the study will be up to 2 years from the time of enrollment.

Target Accrual:

This is a multi-site study in which it is anticipated approximately 28 to 55 participants will be enrolled in order to achieve treatment of approximately 100 bleeding events. The study will target enrollment of a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments.

Data Analysis:

Sample Size Determination:

Results from the Haven 1 study were used to calculate the annual bleeding rates (ABR) in individuals receiving prophylactic treatment. These were calculated at an ABR of three for participants receiving emicizumab prophylactic treatment and 20 for participants receiving other treatments.

Based on these data, it was calculated that between 28 and 55 participants would be necessary to reach 100 BEs with a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments.

Analysis Populations:

The Safety Analysis Set is defined as all participants who received at least a single dose of SEVENFACT®. All analyses of safety will be performed based on the safety population, and participants will be analyzed according to the dose of SEVENFACT® that they actually received.

Baseline Characteristics:

Baseline characteristics will be summarized using descriptive statistics for continuous variables, and frequencies and percentages for categorical variables.

Safety Evaluations:

All Adverse Events (AEs) will be graded for severity utilizing Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and coded using Medical Dictionary of Regulatory Activities (MedDRA) version 23.x. The number and percentage of participants with treatment-emergent AEs (TEAEs), serious AEs (SAEs), serious TEAEs and treatment related TEAEs (i.e., adverse drug reactions [ADRs]) will be presented for all participants.

The number of TEAEs, as well as the number and percentage of participants with TEAEs, serious TEAEs, and treatment-related TEAEs will be presented by MedDRA System Organ Class (SOC) and preferred term for all participants.

The number and percentage of participants with treatment-emergent adverse event and/or allergic and anaphylactic reactions will be presented for all participants.

Efficacy Evaluations:

There are no pre-specified efficacy endpoints.

Study Type

Interventional

Enrollment (Estimated)

55

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Carol Fedor, ND, RN, CCRC
  • Phone Number: 122 (800)-360-2846
  • Email: cfedor@athn.org

Study Contact Backup

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Center for Bleeding Disorders
        • Contact:
        • Principal Investigator:
          • Shelley Crary, MD
    • California
      • Los Angeles, California, United States, 90007
        • Recruiting
        • Orthopaedic Institute for Children
        • Contact:
        • Principal Investigator:
          • Doris Quon, MD
      • Los Angeles, California, United States, 90027
        • Not yet recruiting
        • Childrens Hospital Los Angeles
        • Contact:
        • Principal Investigator:
          • Guy Young, MD
      • Sacramento, California, United States, 95817
        • Not yet recruiting
        • University of California at Davis UC Davis Hemostasis and Thrombosis Center
        • Contact:
        • Principal Investigator:
          • Adam Giermasz, MD
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Recruiting
        • Children's National Hemophilia Center
        • Contact:
          • Michael Guerrera, MD
        • Principal Investigator:
          • Michael Guerrera, MD
    • Florida
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders
        • Contact:
        • Principal Investigator:
          • Shveta Gupta, MD
    • Georgia
      • Atlanta, Georgia, United States, 31404
        • Recruiting
        • Hemophilia of Georgia Center for Bleeding and Clotting Disorders of Emory, Adult Division
        • Principal Investigator:
          • Megan Brown, MD
        • Contact:
      • Savannah, Georgia, United States, 31404
        • Recruiting
        • Willett Children's Hospital at Memorial University Medical Center
        • Contact:
        • Principal Investigator:
          • Ashley Eason
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Withdrawn
        • Louisiana Center for Bleeding and Clotting Disorders, Tulane
      • Slidell, Louisiana, United States, 70461
        • Recruiting
        • Louisiana Center for Advanced Medicine
        • Contact:
        • Principal Investigator:
          • Sharon Pennington, MD
    • Maine
      • Scarborough, Maine, United States, 04074
        • Withdrawn
        • Maine Hemophilia and Thrombosis Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
        • Contact:
        • Principal Investigator:
          • Eric Grabowski, MD
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Health System
        • Contact:
        • Principal Investigator:
          • Philip Kuriakose, MD
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Children's Hospital of Michigan
        • Contact:
        • Principal Investigator:
          • Meera Chitlur, MD
      • Lansing, Michigan, United States, 48912
        • Recruiting
        • MSU Center for Bleeding and Clotting Disorders
        • Contact:
        • Principal Investigator:
          • Shawn Jobe, MD
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Not yet recruiting
        • Center for Bleeding and Clotting Disorders, University of Minnesota
        • Contact:
          • Mark Reding, MD
        • Principal Investigator:
          • Mark Reding, MD
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Comprehensive Hemophilia Center
        • Contact:
        • Principal Investigator:
          • Rajiv Pruthi, MD
    • Mississippi
      • Madison, Mississippi, United States, 39110
        • Recruiting
        • Mississippi Center for Advanced Medicine
        • Contact:
        • Principal Investigator:
          • Spencer Sullivan, MD
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Kansas City Regional Hemophilia Center
        • Contact:
        • Principal Investigator:
          • Lauren Amos, MD
    • New York
      • New Hyde Park, New York, United States, 11040
        • Recruiting
        • Northwell Health, Long Island Jewish
        • Contact:
        • Principal Investigator:
          • Suchitra Acharya, MD
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • Not yet recruiting
        • Brody School of Medicine at East Carolina University
        • Contact:
          • Darla Liles
        • Principal Investigator:
          • Darla Liles, MD
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • University Hospitals Health System Cleveland
        • Contact:
        • Principal Investigator:
          • Sanjay Ahuja, MD
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Not yet recruiting
        • Oklahoma Center for Bleeding and Clotting Disorders
        • Contact:
        • Principal Investigator:
          • Osman Khan, MD
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
        • Principal Investigator:
          • Allison Wheeler, MD
        • Sub-Investigator:
          • Michelle Chi, MD
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Gulf States Hemophilia and Thrombophilia Center-University of Texas Health Science Center @Houston
        • Contact:
        • Principal Investigator:
          • Miguel Escobar, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Have a diagnosis of hemophilia A or B with inhibitors.
  2. Be 12 years of age and older
  3. 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
  4. Have read, understood, and documented written informed consent/assent
  5. Be able to provide medical evidence through prior medical history of previous inhibitor levels
  6. Be willing and able to use the ATHN mobile application or a paper diary to document BEs and medication usage

Exclusion Criteria:

  1. Have a disorder of hemostasis in addition to Hemophilia A or B
  2. Have a known or suspected intolerance or hypersensitivity to SEVENFACT® or its ingredients
  3. Have a known allergy or hypersensitivity to rabbits or rabbit proteins
  4. Are receiving prophylactic treatment for bleeding with a drug or biologic that is not approved for this use by the FDA
  5. Have had implantation of an investigational medical device within the prior 6 months
  6. Have received an investigational drug within 30 days of the baseline visit
  7. Have an elective surgical procedure planned during the duration of their participation in the study*

  8. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Hemophilia A and B Cases
SEVENFACT® 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.
Drug: coagulation factor VIIa [recombinant]-jncw
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.
Other Names:
  • SEVENFACT

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants and percentage of Safety Events (AEs)
Time Frame: From time of consent through BE onset until 3 days after last dose of SEVENFACT®.
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®.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

American Thrombosis and Hemostasis Network

Collaborators

LFB USA, Inc.

Investigators

  • Principal Investigator: Mark Reding, MD, University of Minnesota
  • Principal Investigator: Tammuella Chrisentery-Singleton, MD, American Thrombosis and Hemostasis Network

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 28, 2021

Primary Completion (Estimated)

November 30, 2024

Study Completion (Estimated)

June 1, 2025

Study Registration Dates

First Submitted

November 23, 2020

First Submitted That Met QC Criteria

November 23, 2020

First Posted (Actual)

November 30, 2020

Study Record Updates

Last Update Posted (Estimated)

November 21, 2023

Last Update Submitted That Met QC Criteria

November 20, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATHN 16

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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