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SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

Primary Purpose

Hemophilia A With Inhibitor, Hemophilia B With Inhibitor

Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
coagulation factor VIIa [recombinant]-jncw
Sponsored by
American Thrombosis and Hemostasis Network
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A With Inhibitor focused on measuring Hemophilia A, Hemophilia B, bleeding event, prophylactic treatment

Eligibility Criteria

12 Years - 100 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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    • Should a participant require an unplanned surgery, the participant will not be withdrawn from the study unless the investigator deems it necessary.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.

Sites / Locations

  • Arizona Hemophilia and Thrombosis Center at Phoenix Children's HospitalRecruiting
  • Arkansas Center for Bleeding DisordersRecruiting
  • Orthopaedic Institute for ChildrenRecruiting
  • Childrens Hospital Los Angeles
  • University of California at Davis UC Davis Hemostasis and Thrombosis Center
  • Children's National Hemophilia CenterRecruiting
  • Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood DisordersRecruiting
  • Hemophilia of Georgia Center for Bleeding and Clotting Disorders of Emory, Adult DivisionRecruiting
  • Willett Children's Hospital at Memorial University Medical CenterRecruiting
  • Louisiana Center for Bleeding and Clotting Disorders, Tulane
  • Louisiana Center for Advanced MedicineRecruiting
  • Maine Hemophilia and Thrombosis Center
  • Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment CenterRecruiting
  • Children's Hospital of MichiganRecruiting
  • Henry Ford Health SystemRecruiting
  • MSU Center for Bleeding and Clotting DisordersRecruiting
  • Center for Bleeding and Clotting Disorders, University of Minnesota
  • Mayo Comprehensive Hemophilia CenterRecruiting
  • Mississippi Center for Advanced MedicineRecruiting
  • Kansas City Regional Hemophilia CenterRecruiting
  • Northwell Health, Long Island JewishRecruiting
  • Brody School of Medicine at East Carolina University
  • University Hospitals Health System ClevelandRecruiting
  • Oklahoma Center for Bleeding and Clotting Disorders
  • Vanderbilt University Medical CenterRecruiting
  • Gulf States Hemophilia and Thrombophilia Center-University of Texas Health Science Center @HoustonRecruiting

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Hemophilia A and B Cases

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

First Posted
November 23, 2020
Last Updated
October 4, 2023
Sponsor
American Thrombosis and Hemostasis Network
Collaborators
LFB USA, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04647227
Brief Title
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Official Title
Safety of SEVENFACT® for the Treatment of Bleeding Events in Patients With Hemophilia A or B With Inhibitors
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 28, 2021 (Actual)
Primary Completion Date
November 30, 2024 (Anticipated)
Study Completion Date
June 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
American Thrombosis and Hemostasis Network
Collaborators
LFB USA, Inc.

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Keywords
Hemophilia A, Hemophilia B, bleeding event, prophylactic treatment

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
ATHN 16 is a phase IV multi-center, US-centric, open-label, safety study
Masking
None (Open Label)
Allocation
N/A
Enrollment
55 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Hemophilia A and B Cases
Arm Type
Other
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
coagulation factor VIIa [recombinant]-jncw
Other Intervention Name(s)
SEVENFACT
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of participants and percentage of Safety Events (AEs)
Description
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.
Time Frame
From time of consent through BE onset until 3 days after last dose of SEVENFACT®.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
100 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Carol Fedor, ND, RN, CCRC
Phone
(800)-360-2846
Ext
122
Email
cfedor@athn.org
First Name & Middle Initial & Last Name or Official Title & Degree
Jessica Callis
Phone
800-360-2846
Ext
123
Email
jcallis@athn.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tammuella Chrisentery-Singleton, MD
Organizational Affiliation
Louisiana Center for Advanced Medicine
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Mark Reding, MD
Organizational Affiliation
University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shanna White, MD
Email
swhite@phoenixchildrens.com
First Name & Middle Initial & Last Name & Degree
Erica Sieber, RN
Email
esieber@phoenixchildrens.com
First Name & Middle Initial & Last Name & Degree
Shanna White, MD
Facility Name
Arkansas Center for Bleeding Disorders
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carol Pierce, RN
Phone
501-364-1076
Email
piercecarold@uams.edu
First Name & Middle Initial & Last Name & Degree
Shelley Crary, MD
Facility Name
Orthopaedic Institute for Children
City
Los Angeles
State/Province
California
ZIP/Postal Code
90007
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Doris Quon, MD
Email
dquon@mednet.ucla.edu
First Name & Middle Initial & Last Name & Degree
Doris Quon, MD
Facility Name
Childrens Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elsa Raigoza
Email
eraigoza@chla.usc.edu
First Name & Middle Initial & Last Name & Degree
Guy Young, MD
Facility Name
University of California at Davis UC Davis Hemostasis and Thrombosis Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Adam Giermasz, MD
Email
Giermasz@ucdavis.edu
First Name & Middle Initial & Last Name & Degree
Adam Giermasz, MD
Facility Name
Children's National Hemophilia Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michael Guerrera, MD
First Name & Middle Initial & Last Name & Degree
Michael Guerrera, MD
Facility Name
Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sheila Frederick
Email
sheila.frederick@orlandohealth.com
First Name & Middle Initial & Last Name & Degree
Shveta Gupta, MD
Facility Name
Hemophilia of Georgia Center for Bleeding and Clotting Disorders of Emory, Adult Division
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
31404
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Glaivy Batsuli, MD
Email
gbatsul@emory.edu
First Name & Middle Initial & Last Name & Degree
Megan Brown, MD
Facility Name
Willett Children's Hospital at Memorial University Medical Center
City
Savannah
State/Province
Georgia
ZIP/Postal Code
31404
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alta Castellano, RN
Email
Alta.Castellino@hcahealthcare.com
First Name & Middle Initial & Last Name & Degree
Ashley Eason
Facility Name
Louisiana Center for Bleeding and Clotting Disorders, Tulane
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Individual Site Status
Withdrawn
Facility Name
Louisiana Center for Advanced Medicine
City
Slidell
State/Province
Louisiana
ZIP/Postal Code
70461
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jesus Monico
Email
jmonico@msadvancedmedicine.com
First Name & Middle Initial & Last Name & Degree
Sharon Pennington, MD
Facility Name
Maine Hemophilia and Thrombosis Center
City
Scarborough
State/Province
Maine
ZIP/Postal Code
04074
Country
United States
Individual Site Status
Withdrawn
Facility Name
Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carmen Zhou
Phone
617-726-2737
Email
czhou12@mgh.harvard.edu
First Name & Middle Initial & Last Name & Degree
Eric Grabowski, MD
Facility Name
Children's Hospital of Michigan
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Meera Chitlur, MD
Email
mchitlur@dmc.org
First Name & Middle Initial & Last Name & Degree
Meera Chitlur, MD
Facility Name
Henry Ford Health System
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philip Kuriakose, MD
Email
pkuriak1@hfhs.org
First Name & Middle Initial & Last Name & Degree
Philip Kuriakose, MD
Facility Name
MSU Center for Bleeding and Clotting Disorders
City
Lansing
State/Province
Michigan
ZIP/Postal Code
48912
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anna Burghardt
Email
annarn@msu.edu
First Name & Middle Initial & Last Name & Degree
Shawn Jobe, MD
Facility Name
Center for Bleeding and Clotting Disorders, University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mark Reding, MD
First Name & Middle Initial & Last Name & Degree
Mark Reding, MD
Facility Name
Mayo Comprehensive Hemophilia Center
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rajiv Pruthi, MD
Email
pruthi.rajiv@mayo.edu
First Name & Middle Initial & Last Name & Degree
Rajiv Pruthi, MD
Facility Name
Mississippi Center for Advanced Medicine
City
Madison
State/Province
Mississippi
ZIP/Postal Code
39110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Wendy Thomson
Email
wthomson@msadvancedmedicine.com
First Name & Middle Initial & Last Name & Degree
Spencer Sullivan, MD
Facility Name
Kansas City Regional Hemophilia Center
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eryn Bilynsky
Phone
816-302-6853
Email
erbilynsky@cmh.edu
First Name & Middle Initial & Last Name & Degree
Lauren Amos, MD
Facility Name
Northwell Health, Long Island Jewish
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Suchitra Acharya, MD
Email
Sacharya@northwell.edu
First Name & Middle Initial & Last Name & Degree
Suchitra Acharya, MD
Facility Name
Brody School of Medicine at East Carolina University
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Darla Liles
First Name & Middle Initial & Last Name & Degree
Darla Liles, MD
Facility Name
University Hospitals Health System Cleveland
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sally Muehle
Phone
216-983-3178
Email
sally.muehle@uhhospitals.org
First Name & Middle Initial & Last Name & Degree
Sanjay Ahuja, MD
Facility Name
Oklahoma Center for Bleeding and Clotting Disorders
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Osman Khan, MD
Email
osman-khan@ouhsc.edu
First Name & Middle Initial & Last Name & Degree
Osman Khan, MD
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alyssa Weakley
Email
alyssa.weakley@vumc.org
First Name & Middle Initial & Last Name & Degree
Allison Wheeler, MD
First Name & Middle Initial & Last Name & Degree
Michelle Chi, MD
Facility Name
Gulf States Hemophilia and Thrombophilia Center-University of Texas Health Science Center @Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Miguel Escobar, MD
Email
Miguel.Escobar@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Miguel Escobar, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

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