Emicizumab in Acquired Hemophilia A

NCT04188639 | Completed Phase 2 DMC

• 1 other identifier: AHA-EMI (MO41153)
• Study Type: interventional
• Target: 47
• Locations: 2 countries
• Sites: 16

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).

Conditions

Hemophilia A, Acquired

Keywords

acquired Hemophilia A; Factor VIII activity; Emicizumab

Outcome Measures

Primary Outcomes (1)

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

  12 weeks

Secondary Outcomes (6)

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

  12 weeks

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

  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

  24 weeks

• Days in hospital during 12 weeks of emicizumab treatment

  12 weeks

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

  24 weeks

Study Arms (1)

Treatment with emicizumab

EXPERIMENTAL

Drug: Emicizumab Injection

Interventions

Emicizumab Injection DRUG

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.

Also known as: Hemlibra (R)

Treatment with emicizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• GWT-TUD GmbH: lead
• Hoffmann-La Roche: collaborator
• Hannover Medical School: collaborator

Study Sites (16)

Medizinische Universität Wien, Hämatologie/Hämostaseologie

Wien, Lower Austria, 1090, Austria

Location

Medizinische Universitätsklinik Graz

Graz, Styria, 8036, Austria

Location

Landeskrankenhaus Salzburg, Universitätsklinikum der PMU, Innere Med. III

Salzburg, 5020, Austria

Location

LMU Klinikum, Hämophiliezentrum Erwachsene/Transfusionsmedizin

München, Bavaria, 80336, Germany

Location

Universitätsklinikum Regensburg, Innere Med. III - Studienzentrale

Regensburg, Bavaria, 93052, Germany

Location

Universitätsklinikum Frankfurt, Hämostaseologie/Hämophiliezentrum

Frankfurt am Main, Hesse, 60590, Germany

Location

Universitätsklinikum Gießen und Marburg

Giessen, Hesse, 35392, Germany

Location

Medizinische Hochschule Hannover, Hämatologie/Hämostaseologie

Hanover, Lower Saxony, 30625, Germany

Location

Universitätsklinikum Bonn, Hämatologie/Transfusionsmedizin/Hämophilie

Bonn, North Rhine-Westphalia, 53127, Germany

Location

Universitätsklinikum des Saarlandes, Institut für Klinische Hämostaseologie und Transfusionsmedizin

Homburg / Saar, Saarland, 66421, Germany

Location

Universitätsklinikum Dresden, Med. Poliklinik I

Dresden, Saxony, 01307, Germany

Location

Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I, Bereich Hämostaseologie

Leipzig, Saxony, 04103, Germany

Location

Universitätsklinikum Schleswig-Holstein, Klinische Chemie/Gerinnungszentrum

Kiel, Schleswig-Holstein, 24105, Germany

Location

Vivantes Klinikum im Friedrichshain, Angiologie/Hämostaseologie

Friedrichshain, State of Berlin, 10249, Germany

Location

Universitätsklinikum Jena, Klinik für Innere Medizin II

Jena, Thuringia, 07747, Germany

Location

Universitätsklinikum Hamburg-Eppendorf, Med. Klinik II/Gerinnungsambulanz

Hamburg, 20246, Germany

Location

Related Publications (2)

• 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.

  PMID: 40795229 DERIVED

• 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.

  PMID: 37858328 DERIVED

MeSH Terms

Conditions

Factor 8 deficiency, acquired

Interventions

emicizumab

Study Officials

• Andreas Tiede, Prof. Dr.

  Hannover Medical School

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2019
• First Posted: December 6, 2019
• Study Start: March 23, 2021
• Primary Completion: January 4, 2023
• Study Completion: January 4, 2023
• Last Updated: January 9, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); DE (13)