Emicizumab in Patients With Acquired Hemophilia A (AHAEmi)

November 3, 2025 updated by: Rebecca Kruse-Jarres, University of Washington

Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial

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).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

51

Phase

  • Phase 2

Contacts and Locations

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

Study Locations

  • United States
    • California
      • San Diego, California, United States, 92121
        • UCSD Hemophilia and Thrombosis Treatment Center
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Georgetown University
    • Georgia
      • Atlanta, Georgia, United States, 30308
        • Emory University
    • Illinois
      • Peoria, Illinois, United States, 61614
        • Bleeding and Clotting Disorders Institute
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center, Inc.
    • Louisiana
      • New Orleans, Louisiana, United States, 70112-2632
        • Tulane University
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • University of North Carolina
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma Health Sciences Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Penn Blood Disorders Program, Hospital of the University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Hemophilia Center of Western Pennsylvania
    • Vermont
      • Burlington, Vermont, United States, 05401
        • University of Vermont Medical Center
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • UVA Comprehensive Cancer Center
    • Washington
      • Seattle, Washington, United States, 98101
        • Washington Center for Bleeding Disorders
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Versiti Inc.

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

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

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
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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Primary Outcome Meassure
Time Frame: 12 weeks
Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence and severity of adverse events
Time Frame: 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.
duration of entire study
Days of treatment with additional hemostatic agent
Time Frame: 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
12 weeks
Remission Rate
Time Frame: 1 to 24 weeks
Number of patients achieving partial remission (PR) and complete remission (CR) over 12 and 24 weeks after starting emicizumab prophylaxis
1 to 24 weeks
Hospitalizations
Time Frame: 12 weeks
Days in hospital during week 12 of emicizumab treatment
12 weeks
Total dose of additional hemostatic agent
Time Frame: 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
12 weeks

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Comparison of historic GTH-AH 01/2020 cohort/ Number of clinically significant bleeds
Time Frame: 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
12 to 24 weeks
Comparison to a parallel German study cohort/ Number of clinically significant bleeds
Time Frame: 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
12 to 24 weeks

Collaborators and Investigators

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

Sponsor

University of Washington

Collaborators

Genentech, Inc.

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

August 31, 2022

Primary Completion (Estimated)

July 1, 2026

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

April 18, 2022

First Submitted That Met QC Criteria

April 22, 2022

First Posted (Actual)

April 25, 2022

Study Record Updates

Last Update Posted (Estimated)

November 5, 2025

Last Update Submitted That Met QC Criteria

November 3, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STUDY00013920

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

All of the individual participant data collected during the trial will be shared with the research group of the parallel European study (NCT04188639). Data will be shared at the end of the study for analysis for a period of 2 years.

IPD Sharing Time Frame

Data will be shared with the research group of the parallel European study (NCT04188639) at the end of the study and will be available for 2 years.

IPD Sharing Access Criteria

Only researchers of this or the parallel trial (NCT04188639) will have access to this data.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

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.

No

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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