Evaluation of the effect of emicizumab in patients suffering from acute bleeds with previously no family history.

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 47

Inclusion Criteria

1)Patients diagnosed with AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) at the time of diagnosis (local laboratory)
2)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
3)Male or female patients aged 18 years or older at the time of informed consent
4)Ability to understand and follow study-related instructions
5)Current bleeds due to AHA at the time of screening

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 23

Exclusion Criteria

1)Congenital hemophilia A
2) Partial or complete remission of AHA (defined as FVIII:C =50% and no bleeding and no hemostatic therapy) at the time of screening
3)Treatment with aPCC within the last 48 h before first study treatment or planned treatment with aPCC during the course of the study
4)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 discretion.
5)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
6)Positive lupus anticoagulant at the time of screening
7)Severe uncontrolled infection at the time of screening
8)Signs of active disseminated intravascular coagulation at the time of screening
9)Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
10)Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator’s judgment
11)Known severe congenital or acquired thrombophilia
12)Life expectancy <3 months at the time of screening
13)Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
14)Contraindications according to the Investigator’s Brochure of emicizumab
15)Current treatment with emicizumab at time of screening
16)History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
17)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
18)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
19)Pregnant or breast-feeding women
20)Women of childbearing potential unless women who meet the following criteria:
a.Post-menopausal (12 months natural amenorrhea or 6 months amenorrhea with serum FSH > 40 U/mL)
b.Postoperatively (six weeks after bilateral ovariectomy with or without hysterectomy)
c.Regular and correct use of a contraceptive method with error rate <1% per year such as implants, depot injections, oral contraceptives or intrauterine devices
d.Sexual abstinence
e.Vasectomy of the partner
21)Subject is in custody by order of an authority or a court of law
22)Receipt of an investigational drug concurrently or within 5 half-lives before administration of the study drug

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the present study is to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).;Secondary Objective: - To study the safety of emicizumab in patients with AHA<br>- To compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort<br>- To compare bleeding and adverse events with a parallel US study<br>- To evaluate the pharmacokinetics (PK) of emicizumab in patients with AHA<br><br>;Primary end point(s): The primary efficacy objective is to evaluate the efficacy of prophylactic emicizumab which will be evaluated by determination of the rate of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first.;Timepoint(s) of evaluation of this end point: 12 weeks

Secondary Outcome Measures

Name	Time	Method

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