Emicizumab in Acquired Hemophilia A

January 6, 2023 updated by: GWT-TUD GmbH

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
47

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

  • Austria
      • Salzburg, Austria, 5020
        • Landeskrankenhaus Salzburg, Universitätsklinikum der PMU, Innere Med. III
    • Niederösterreich
      • Wien, Niederösterreich, Austria, 1090
        • Medizinische Universität Wien, Hämatologie/Hämostaseologie
    • Steiermark
      • Graz, Steiermark, Austria, 8036
        • Medizinische Universitätsklinik Graz
  • Germany
      • Hamburg, Germany, 20246
        • Universitätsklinikum Hamburg-Eppendorf, Med. Klinik II/Gerinnungsambulanz
    • Bayern
      • München, Bayern, Germany, 80336
        • LMU Klinikum, Hämophiliezentrum Erwachsene/Transfusionsmedizin
      • Regensburg, Bayern, Germany, 93052
        • Universitätsklinikum Regensburg, Innere Med. III - Studienzentrale
    • Berlin
      • Berlin-Friedrichshain, Berlin, Germany, 10249
        • Vivantes Klinikum im Friedrichshain, Angiologie/Hämostaseologie
    • Hessen
      • Frankfurt, Hessen, Germany, 60590
        • Universitätsklinikum Frankfurt, Hämostaseologie/Hämophiliezentrum
      • Gießen, Hessen, Germany, 35392
        • Universitätsklinikum Gießen und Marburg
    • Niedersachsen
      • Hannover, Niedersachsen, Germany, 30625
        • Medizinische Hochschule Hannover, Hämatologie/Hämostaseologie
    • Nordrhein-Westfalen
      • Bonn, Nordrhein-Westfalen, Germany, 53127
        • Universitätsklinikum Bonn, Hämatologie/Transfusionsmedizin/Hämophilie
    • Saarland
      • Homburg / Saar, Saarland, Germany, 66421
        • Universitätsklinikum des Saarlandes, Institut für Klinische Hämostaseologie und Transfusionsmedizin
    • Sachsen
      • Dresden, Sachsen, Germany, 01307
        • Universitätsklinikum Dresden, Med. Poliklinik I
      • Leipzig, Sachsen, Germany, 04103
        • Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I, Bereich Hämostaseologie
    • Schleswig-Holstein
      • Kiel, Schleswig-Holstein, Germany, 24105
        • Universitätsklinikum Schleswig-Holstein, Klinische Chemie/Gerinnungszentrum
    • Thüringen
      • Jena, Thüringen, Germany, 07747
        • Universitätsklinikum Jena, Klinik für Innere Medizin II

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients diagnosed with AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) (local laboratory) at time of diagnosis
  • 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
  • Partial or complete remission of AHA (defined as FVIII activity ≥ 50 % and no bleeding and no hemostatic therapy) at the time of screening
  • 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 immunosuppressive 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

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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 follow-up with Immunosuppressive therapy (IST) at the investigators discretion.
Other Names:
  • Hemlibra (R)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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
12 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab
Time Frame: 12 weeks
12 weeks
Incidence of mortality and cause of death in the 24 weeks after starting emicizumab treatment
Time Frame: 24 weeks
24 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
Time Frame: 24 weeks
24 weeks
Days in hospital during 12 weeks of emicizumab treatment
Time Frame: 12 weeks
12 weeks
Number of patients achieving partial remission in the 24 weeks after starting emicizumab treatment
Time Frame: 24 weeks
24 weeks
Bleeding-free survival in the 12 weeks after starting emicizumab treatment
Time Frame: 12 weeks
12 weeks

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
GWT-TUD GmbH

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Hannover Medical School
Hoffmann-La Roche

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Andreas Tiede, Prof. Dr., Hannover Medical School

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 23, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 4, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 4, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 4, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 5, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 6, 2019

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 9, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 6, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AHA-EMI (MO41153)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

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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