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Emicizumab in Acquired Hemophilia A

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ClinicalTrials.gov Identifier: NCT04188639
Recruitment Status : Recruiting
First Posted : December 6, 2019
Last Update Posted : August 3, 2021
Sponsor:
Collaborators:
Hoffmann-La Roche
Hannover Medical School
Information provided by (Responsible Party):
GWT-TUD GmbH

Study Description
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Brief Summary:
This study is an international, multicenter, open-label, single arm, prospective clinical trial and will 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
Hemophilia A, Acquired Drug: Emicizumab Injection Phase 2

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 : March 23, 2021
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : September 2024

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: Treatment with emicizumab Drug: Emicizumab Injection
All eligible patients with AHA will receive the same study medication consisting of once weekly subcutaneous emicizumab. For each subject, the maximal duration of the study will be 24 weeks including 12 weeks treatment with emicizumab and 12 weeks Immunosuppressive therapy according to local standard of care.
Other Name: Hemlibra (R)


Outcome Measures
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Primary Outcome Measures :
  1. The number of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab [ Time Frame: 12 weeks ]
  2. Incidence of mortality and cause of death in the 24 weeks after starting emicizumab treatment [ Time Frame: 24 weeks ]
  3. 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 [ Time Frame: 24 weeks ]
  4. Days in hospital during 12 weeks of emicizumab treatment [ Time Frame: 12 weeks ]
  5. Number of patients achieving partial remission in the 24 weeks after starting emicizumab treatment [ Time Frame: 24 weeks ]

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:

  • Patients diagnosed with AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) at screening (local laboratory)
  • Signed informed consent form by the participant or a Person who is legally authorized to sign on behalf of the participant before any study specific tests or procedures are done
  • Male or female patients aged 18 years or older at the time of informed consent
  • Ability to understand and follow study-related instructions
  • Current bleeds due to AHA at the time of screening

Exclusion Criteria:

  • Congenital hemophilia A
  • Treatment with aPCC within the last 48 h before first study treatment or planned treatment with aPCC during the course of the study
  • Treatment of AHA within the days before study enrollment with more than 100 mg prednisolone (or equivalent) per day or prednisolone for more than 2 days or with other immunosuppressive drugs (e.g. rituximab, cyclophosphamide). IST for other concomitant disorders (e.g. autoimmune disorders) is not an exclusion criterion and can be continued at the investigator's discrétion
  • Therapy (current or planned during the emicizumab treatment period) with immunospuppressive or immune modulating drugs that were not already given on a regular basis before first diagnosis of AHA
  • 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
  • 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 local SmPC of emicizumab (see 16.1 Appendix I)
  • 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

  • Women of childbearing potential unless women who meet the following criteria:

    1. Post-menopausal (12 months natural amenorrhea or 6 months amenorrhea with serum FSH > 40 U/mL)
    2. Postoperatively (six weeks after bilateral ovariectomy with or without hysterectomy)
    3. Regular and correct use of a contraceptive method with error rate <1% per year such as implants, depot injections, oral contraceptives or intrauterine devices
    4. Sexual abstinence
    5. Vasectomy of the partner
  • Men of sexual activity with women of childbearing potential who are not willing to use an effective barrier method of contraception during and up to 3 months after the end of therapy
  • Subject is in custody by order of an authority or a court of law
  • Receipt of an investigational drug concurrently or within 5 half-lives before administration of the study drug
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): NCT04188639


Contacts
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Contact: GWT-TUD GmbH +49 (0) 351 25933 100 medical.consulting@gwtonline.de
Contact: Carmen Weigt, Dr. carmen.weigt@gwtonline.de

Locations
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Germany
LMU Klinikum Recruiting
München, Bayern, Germany, 80336
Contact: Patrick Möhnle, Dr.    +49 89 4400 57696    patrick.moehnle@med.uni-muenchen.de   
Vivantes Klinikum im Friedrichshain Recruiting
Berlin-Friedrichshain, Berlin, Germany, 10249
Contact: Robert Klamroth, PD Dr.    +49 (0) 30 130 23 1575    robert.klamroth@vivantes.de   
Medizinische Hochschule Hannover Recruiting
Hannover, Niedersachsen, Germany, 30625
Contact: Susanne Bartels    +49(0) 511 532 ext 3636    tiede.clinical-trials@mh-hannover.de   
Principal Investigator: Andreas Tiede, Prof. Dr. med.         
Sub-Investigator: Christiane Dobbelstein, Dr. med.         
Universitätsklinikum des Saarlandes, Institut für Klinische Hämostaseologie und Transfusionsmedizin Recruiting
Homburg / Saar, Saarland, Germany, 66421
Contact: Hermann Eichler, Prof.    +49 (0)6841 1622530    hermann.eichler@uks.eu   
Universitätsklinikum Dresden Recruiting
Dresden, Sachsen, Germany, 01307
Contact: Karolin Trautmann-Grill, Dr.    +49 351 458 2311    karolin.trautmann@ukdd.de   
Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I, Bereich Hämostaseologie Recruiting
Leipzig, Sachsen, Germany, 04103
Contact: Christian Pfrepper, Dr.    + 49 341 97 12700    christian.pfrepper@medizin.uni-leipzig.de   
Universitätsklinikum Jena, Klinik für Innere Medizin II Not yet recruiting
Jena, Thüringen, Germany, 07747
Contact: Krisitna Schilling, Dr.    +49 (0) 3641 932 45 72    Kristina.Schilling@med.uni-jena.de   
Sponsors and Collaborators
GWT-TUD GmbH
Hoffmann-La Roche
Hannover Medical School
Investigators
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Study Director: Andreas Tiede, Prof. Dr. Hannover Medical School
More Information
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Responsible Party: GWT-TUD GmbH
ClinicalTrials.gov Identifier: NCT04188639    
Other Study ID Numbers: AHA-EMI (MO41153)
First Posted: December 6, 2019    Key Record Dates
Last Update Posted: August 3, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by GWT-TUD GmbH:
acquired Hemophilia A
Factor VIII activity
Emicizumab
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