SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

  • STATUS
    Recruiting
  • End date
    Jun 1, 2025
  • participants needed
    55
  • sponsor
    American Thrombosis and Hemostasis Network
Updated on 30 May 2022

Summary

Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors aged 12 to 65 years, inclusive, 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.

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 inhibitors aged 12 to 65 years, inclusive, 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.

Details
Condition Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Treatment coagulation factor VIIa [recombinant]-jncw
Clinical Study IdentifierNCT04647227
SponsorAmerican Thrombosis and Hemostasis Network
Last Modified on30 May 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Have a diagnosis of hemophilia A or B with inhibitors
Be 12 - 65 years of age inclusive
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
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