Emicizumab in Acquired Hemophilia A

• ClinicalTrials.gov Identifier: NCT04188639
• Recruitment Status: Not yet recruiting
• First Posted: December 6, 2019
• Last Update Posted: December 9, 2019
• Sponsor: GWT-TUD GmbH
• Collaborators: Hoffmann-La Roche; Hannover Medical School
• Information provided by (Responsible Party): GWT-TUD GmbH

Study Description

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

• 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
• Estimated Study Start Date: May 2020
• Estimated Primary Completion Date: August 2022
• Estimated Study Completion Date: November 2022

Arms and Interventions

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

Primary Outcome Measures :

1. The number of clinically significant bleeds during emicizumab treatment [ Time Frame: 12 weeks ]

Secondary Outcome Measures :

1. Incidence and severity of adverse events [ Time Frame: 24 weeks ]
   Incidence and severity of adverse events
2. Incidence and severity of thromboembolic events [ Time Frame: 24 weeks ]
   Incidence and severity of thromboembolic events
3. Incidence of mortality [ Time Frame: 24 weeks ]
4. The number of patients with additional Immunosuppressive therapy (IST) [ Time Frame: 12 weeks ]
   The number of patients, in whom IST was started during the 12 weeks of emicizumab prophylaxis because of ≥2 clinically significant bleeds

Eligibility Criteria

• 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 before any study specific tests or procedures
• 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
• 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

Contacts

Contact: GWT-TUD GmbH; +49 (0) 351 25933 100; medical.consulting@gwtonline.de

Contact: Carmen Weigt, Dr.; carmen.weigt@gwtonline.de

Sponsors and Collaborators

GWT-TUD GmbH

Hoffmann-La Roche

Hannover Medical School

Investigators

• Study Director: Andreas Tiede, Prof. Dr.; Hannover Medical School

More Information

• Responsible Party: GWT-TUD GmbH
• ClinicalTrials.gov Identifier: NCT04188639
• Other Study ID Numbers: AHA-EMI (MO41153)
• First Posted: December 6, 2019
• Last Update Posted: December 9, 2019
• Last Verified: December 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• 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:

Hemophilia A; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn