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Hemophilia A, AcquiredPhase 2NCT04188639

Emicizumab in Acquired Hemophilia A

A Phase 2 study testing Emicizumab Injection in people with Hemophilia A, Acquired, run by GWT-TUD GmbH at 16 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Hemophilia A, Acquired
What is being tested?
Emicizumab Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
47 participants
Where
16 sitesin 2 countries
Study dates
23 Mar 2021to 4 Jan 2023
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 28Show fewer
  • 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:
  • Post-menopausal (12 months natural amenorrhea or 6 months amenorrhea with serum FSH > 40 U/mL)
  • Postoperatively (six weeks after bilateral ovariectomy with or without hysterectomy)
  • Regular and correct use of a contraceptive method with error rate <1% per year such as implants, depot injections, oral contraceptives or intrauterine devices
  • Sexual abstinence
  • 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

Trial locations and contacts

16 sites in 2 countries.

16 sites in 2 countries

Germany13 sites

  • Universitätsklinikum Bonn, Hämatologie/Transfusionsmedizin/Hämophilie

    Bonn, North Rhine-Westphalia, 53127

  • Universitätsklinikum Dresden, Med. Poliklinik I

    Dresden, Saxony, 01307

  • Universitätsklinikum Frankfurt, Hämostaseologie/Hämophiliezentrum

    Frankfurt am Main, Hesse, 60590

  • Vivantes Klinikum im Friedrichshain, Angiologie/Hämostaseologie

    Friedrichshain, State of Berlin, 10249

  • Universitätsklinikum Gießen und Marburg

    Giessen, Hesse, 35392

  • Universitätsklinikum Hamburg-Eppendorf, Med. Klinik II/Gerinnungsambulanz

    Hamburg, 20246

  • Medizinische Hochschule Hannover, Hämatologie/Hämostaseologie

    Hanover, Lower Saxony, 30625

  • Universitätsklinikum des Saarlandes, Institut für Klinische Hämostaseologie und Transfusionsmedizin

    Homburg / Saar, Saarland, 66421

  • Universitätsklinikum Jena, Klinik für Innere Medizin II

    Jena, Thuringia, 07747

  • Universitätsklinikum Schleswig-Holstein, Klinische Chemie/Gerinnungszentrum

    Kiel, Schleswig-Holstein, 24105

  • Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I, Bereich Hämostaseologie

    Leipzig, Saxony, 04103

  • LMU Klinikum, Hämophiliezentrum Erwachsene/Transfusionsmedizin

    München, Bavaria, 80336

  • Universitätsklinikum Regensburg, Innere Med. III - Studienzentrale

    Regensburg, Bavaria, 93052

Other trials in Germany

Austria3 sites

  • Medizinische Universitätsklinik Graz

    Graz, Styria, 8036

  • Landeskrankenhaus Salzburg, Universitätsklinikum der PMU, Innere Med. III

    Salzburg, 5020

  • Medizinische Universität Wien, Hämatologie/Hämostaseologie

    Wien, Lower Austria, 1090

Other trials in Austria

About this study

Official title: 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).

Being tested
Emicizumab Injection
Keywords
acquired Hemophilia A, Factor VIII activity, Emicizumab
Medical subject headings
Factor 8 deficiency, acquired

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Emicizumab InjectionDrug

    Also called Hemlibra (R)

    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.

Study groups in NCT04188639
Study groupRoleWhat they receive
Treatment with emicizumabExperimentalNot described
Receives: Drug: Emicizumab Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The number of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first

    Measured: 12 weeks

Secondary outcomes (6)

  1. Incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab

    Measured: 12 weeks

  2. Incidence of mortality and cause of death in the 24 weeks after starting emicizumab treatment

    Measured: 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

    Measured: 24 weeks

  4. Days in hospital during 12 weeks of emicizumab treatment

    Measured: 12 weeks

  5. Number of patients achieving partial remission in the 24 weeks after starting emicizumab treatment

    Measured: 24 weeks

  6. Bleeding-free survival in the 12 weeks after starting emicizumab treatment

    Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Dec 2019
  2. Study start23 Mar 2021
  3. Primary completion4 Jan 2023
  4. Study completion4 Jan 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
47 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04188639
Sponsor's protocol ID
AHA-EMI (MO41153)
Status last verified
Jan 2023
First submitted
4 Dec 2019
Last updated
9 Jan 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Turkantoz H, Dobbelstein C, Greil R, Hart C, Klamroth R, Knobl P, Oldenburg J, Pfrepper C, Sachs U, Schimansky IM, Trautmann-Grill K, Tiede A. Management of Breakthrough Bleeding During Emicizumab Prophylaxis in Acquired Haemophilia A: Data From the GTH-AHA-EMI Study. Haemophilia. 2026 Jun 15. doi: 10.1111/hae.70339. Online ahead of print.

    PubMed 42290320

  • Schimansky IM, Dobbelstein C, Klamroth R, Hart C, Sachs UJ, Greil R, Knobl P, Oldenburg J, Miesbach W, Pfrepper C, Trautmann-Grill K, Mohnle P, Holstein K, Eichler H, Werwitzke S, Tiede A. Sustained survival benefit of emicizumab and postponed immunosuppression in acquired hemophilia A. Blood Adv. 2025 Nov 25;9(22):5853-5860. doi: 10.1182/bloodadvances.2025017144.

    PubMed 40795229

  • Tiede A, Hart C, Knobl P, Greil R, Oldenburg J, Sachs UJ, Miesbach W, Pfrepper C, Trautmann-Grill K, Holstein K, Pilch J, Mohnle P, Schindler C, Weigt C, Schipp D, May M, Dobbelstein C, Pelzer FJ, Werwitzke S, Klamroth R. Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study. Lancet Haematol. 2023 Nov;10(11):e913-e921. doi: 10.1016/S2352-3026(23)00280-6. Epub 2023 Oct 16.

    PubMed 37858328

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Emicizumab Injection in people with Hemophilia A, Acquired, run by GWT-TUD GmbH at 16 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04188639, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2023.

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