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AHAEmi: Emicizumab in Patients With Acquired Hemophilia A

Sponsor
University of Washington (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05345197
Collaborator
Genentech, Inc. (Industry)
51
Enrollment
1
Location
1
Arm
31
Anticipated Duration (Months)
1.6
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

Patients with AHA who are eligible will receive two loading doses of the study drug, emicizumab (6mg/kg on day 1 and 3 mg/kg on day 2) followed by once weekly subcutaneous emicizumab (1.5 mg/kg). Immunosuppression will be given concurrently as per investigator discretion. The primary endpoint (bleed rate) will be assessed after 12 weeks on study drug. If partial remission of the AHA has not been achieved, an additional 12 weeks of study drug may be given. A historical cohort and a study conducted in parallel in Germany (NCT04188639) will serve as control groups for evaluation of secondary endpoints provided the study cohort are comparable.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
51 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial
Anticipated Study Start Date :
Jun 1, 2022
Anticipated Primary Completion Date :
Jul 1, 2024
Anticipated Study Completion Date :
Jan 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Experimental-treatment

Treatment with emicizumab

Drug: emicizumab
This study design uses emicizumab as a prophylaxis treatment to prevent bleeding for all participants, bypassing agents (with the exception of aPCC) and treatment of concomitant diseases will be given as clinically indicated. All eligible subjects with AHA will receive the same study medication consisting of: two loading doses of the emicizumab on day 1 and 2 followed by once weekly subcutaneous emicizumab injections. Immunosuppressive therapy (IST) will be given concurrently as per investigator discretion.
Other Names:
  • Hemlibra (R)

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Primary Outcome Meassure [12 weeks]

      Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)

    Secondary Outcome Measures

    1. Incidence and severity of adverse events [duration of entire study]

      Incidence and severity of adverse events, including thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab treatment; mortality and cause of death in the 24 weeks after starting emicizumab treatment.

    2. Days of treatment with additional hemostatic agent [12 weeks]

      Days of treatment with and total dose of bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) or recombinant porcine factor VIII (susoctocag alfa) or other factor VIII concentrates; specifics (drug, amount and timing) of IST started during the 12 weeks of emicizumab prophylaxis

    3. Remission Rate [1 to 24 weeks]

      Number of patients achieving partial remission (PR) and complete remission (CR) over 12 and 24 weeks after starting emicizumab prophylaxis

    4. Hospitalizations [12 weeks]

      Days in hospital during week 12 of emicizumab treatment

    5. Total dose of additional hemostatic agent [12 weeks]

      Total dose of bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) or recombinant porcine factor VIII (susoctocag alfa) or other factor VIII concentrates; specifics (drug, amount and timing) of IST started during the 12 weeks of emicizumab prophylaxis

    Other Outcome Measures

    1. Comparison of historic GTH-AH 01/2020 cohort/ Number of clinically significant bleeds [12 to 24 weeks]

      Number of clinically significant bleeds in the 12 weeks of emicizumab treatment or until achieving partial remission (PR) of AHA, whatever occurs first Incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy, in 12 weeks of treatment and mortality after 24 weeks Bleeding-free survival until week 12 after starting treatment

    2. Comparison to a parallel German study cohort/ Number of clinically significant bleeds [12 to 24 weeks]

      Number of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first Incidence and severity of adverse events, thromboembolic events, in the 12 weeks of emicizumab treatment and mortality after 24 weeks Bleeding-free survival until week 12 of emicizumab treatment

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Signed Informed Consent/Assent Form

    • Age ≥18 years at time of signing Informed Consent Form

    • Ability to comply with the study protocol, in the investigator's judgment

    • Diagnosis of AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) at screening (local laboratory)

    • Current bleeding due to AHA at the time of screening

    • Plan to be adherent to emicizumab prophylaxis during the study

    • For women of childbearing potential who meet the following criteria:

    • Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of <1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.

    Exclusion Criteria:

    • Congenital hemophilia A

    • Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study

    • Known positive lupus anticoagulant at the time of screening

    • Severe uncontrolled infection at the time of screening

    • Signs of active disseminated intravascular coagulation at the time of screening -

    • Emicizumab ⎯ AHA Emi Version 1.0 20

    • Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening

    • Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment

    • Known severe congenital or acquired thrombophilia

    • Life expectancy <3 months at the time of screening

    • Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator

    • Contraindications according to the Investigator's Brochure of emicizumab

    • Current treatment with emicizumab at time of screening

    • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator

    • Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study

    • Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator

    • Pregnant or breast-feeding women

    • Would refuse treatment with blood or blood products, if necessary.

    • Subject is in custody by order of an authority or a court of law

    • Treatment with any of the following:

    • An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1

    • A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives- before Study Day 1, whichever is longer

    • An investigational drug concurrently

    • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Washington Center for Bleeding Disorders Seattle Washington United States 98101

    Sponsors and Collaborators

    • University of Washington
    • Genentech, Inc.

    Investigators

    • Principal Investigator: Rebecca Kruse-Jarres, MD, MPH, University of Washington

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Rebecca Kruse-Jarres, Professor, School of Medicine: Hematology, University of Washington
    ClinicalTrials.gov Identifier:
    NCT05345197
    Other Study ID Numbers:
    • STUDY00013920
    First Posted:
    Apr 25, 2022
    Last Update Posted:
    Apr 25, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Rebecca Kruse-Jarres, Professor, School of Medicine: Hematology, University of Washington
    Acquire Hemophilia A
    emicizumab
    AHA
    Additional relevant MeSH terms:
    Hemophilia A

    Study Results

    No Results Posted as of Apr 25, 2022