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Emicizumab in Patients With Acquired Hemophilia A (AHAEmi)

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ClinicalTrials.gov Identifier: NCT05345197
Recruitment Status : Recruiting
First Posted : April 25, 2022
Last Update Posted : April 17, 2024
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Rebecca Kruse-Jarres, University of Washington

Study Description
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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
Acquired Hemophilia A Drug: emicizumab 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
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Study Type : Interventional  (Clinical Trial)
Estimated 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
Actual Study Start Date : August 31, 2022
Estimated Primary Completion Date : July 2024
Estimated Study Completion Date : January 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Emicizumab

Arms and Interventions
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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 Name: Hemlibra (R)


Outcome Measures
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Primary Outcome Measures :
  1. Primary Outcome Meassure [ Time Frame: 12 weeks ]
    Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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

  4. Hospitalizations [ Time Frame: 12 weeks ]
    Days in hospital during week 12 of emicizumab treatment

  5. 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


Other Outcome Measures:
  1. 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

  2. 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


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05345197


Contacts
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Contact: Danielle Matia, MPH 206-614-1157 ext 1017 danielle.matia@wacbd.org
Contact: Sarah Ruuska, MPH 2066497415 ext 1017 sarah.ruuska@wacbd.org

Locations
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United States, California
UCSD Hemophilia and Thrombosis Treatment Center Recruiting
San Diego, California, United States, 92121
Contact: Ilana Levin    858-657-6434    ivlevin@health.ucsd.edu   
Contact: Isabel Chang    858 657 6437    i3chang@health.ucsd.edu   
Principal Investigator: Annette Von Drygalski, MD         
Sub-Investigator: Jenny Zhou, MD         
Sub-Investigator: John Adamson, MD         
United States, District of Columbia
Georgetown University Recruiting
Washington, District of Columbia, United States, 20007
Contact: Helena Jacobs    202-687-4868    helena.Jacobs@georgetown.edu   
Contact: Michelle Cha    202-687-0117    mjc329@georgetown.edu   
Principal Investigator: Craig Kessler, M.D.         
Sub-Investigator: Gary Kupfer, M.D.         
Sub-Investigator: Pedro Alcedo, M.D.         
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30308
Contact: Stephanie Whitten    404-686-0277    stephanie.whitten@emoryhealthcare.org   
Contact: Lucy Sanchez, MS    404 686 0273    lucy.sanchez@emory.edu   
Principal Investigator: Lorraine Cafuir, MD         
Sub-Investigator: Christine Kempton, MD, MSc         
United States, Illinois
Bleeding and Clotting Disorders Institute Recruiting
Peoria, Illinois, United States, 61614
Contact: Shayla Nunn    309-692-5337 ext 135    shayla@ilbcdi.org   
Contact: Nikki Barclay    309-692-5337 ext 139    nikki@ilbcdi.org   
Principal Investigator: Jonathan C. Roberts, MD         
United States, Indiana
Indiana Hemophilia and Thrombosis Center, Inc. Recruiting
Indianapolis, Indiana, United States, 46260
Contact: James Okungade, MB, BS    317-871-0000 ext 1118    jokungade@ihtc.org   
Contact: Neelam Thukral    317 871-0000 ext 1373    nthukral@ihtc.org   
Principal Investigator: Brandon M. Hardesty, MD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Rebecca Trogstad    507-266-1944    trogstad.rebecca@mayo.edu   
Contact: Jasmine Sexton    (507) 266-1944    sexton.jasmine@mayo.edu   
Principal Investigator: Meera Sridharan, M.d., PhD.         
Sub-Investigator: Rajiv Pruthi, M.B.B.S.         
United States, Washington
Washington Center for Bleeding Disorders Recruiting
Seattle, Washington, United States, 98101
Contact: Danielle M. Matia, MPH    206-614-1157 ext 1016    danielle.matia@wacbd.org   
Contact: Sarah Ruuska, MPH    12066497415 ext 1017    sarah.ruuska@wacbd.org   
Sponsors and Collaborators
University of Washington
Genentech, Inc.
Investigators
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Principal Investigator: Rebecca Kruse-Jarres, MD, MPH University of Washington
More Information
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Publications:

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Responsible Party: Rebecca Kruse-Jarres, Professor, School of Medicine: Hematology, University of Washington
ClinicalTrials.gov Identifier: NCT05345197    
Other Study ID Numbers: STUDY00013920
First Posted: April 25, 2022    Key Record Dates
Last Update Posted: April 17, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
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.
Access Criteria: Only researchers of this or the parallel trial (NCT04188639) will have access to this data.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rebecca Kruse-Jarres, University of Washington:
Acquire Hemophilia A
emicizumab
AHA
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn