EINARMIGE, MULTIZENTRISCHE KLINISCHE PHASE-IIIB-STUDIE ZUR UNTERSUCHUNG DER SICHERHEIT UND VERTRÄGLICHKEIT VON PROPHYLAKTISCHEM EMICIZUMAB BEI HÄMOPHILIE-A-PATIENTEN MIT NEUTRALISIERENDEN FAKTOR-VIII-ANTIKÖRPERN („HEMMKÖRPERN“)

Drug: Emicizumab

Inclusion Criteria:

- As per investigator's judgement, a willingness and ability to comply with scheduled
visits, treatment plans, laboratory tests, and other study procedures, including the
patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an
electronic device or paper

- Aged 12 years or older at the time of informed consent

- Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII

- Documented treatment with bypassing agents or FVIII concentrates in the last 6 months
(on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day
before starting emicizumab

- Adequate hematologic, hepatic, and renal function

- For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use a highly effective contraceptive method with a
failure rate of <1% per year during the treatment period and for at least five
elimination half-lives (24 weeks) after the last dose of emicizumab

Exclusion Criteria:

- Inherited or acquired bleeding disorder other than hemophilia A

- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy
(prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to
enrollment). Patients receiving ITI therapy will be eligible following the completion
of a 72-hour washout period prior to the first emicizumab administration

- History of illicit drug or alcohol abuse within 12 months prior to screening, as per
the investigator's judgment

- High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or
family history of TMA), as per the investigator's judgment

- Previous (in the past 12 months) or current treatment for thromboembolic disease (with
the exception of previous catheter-associated thrombosis for which antithrombotic
treatment is not currently ongoing) or current signs of thromboembolic disease

- Other conditions (e.g., certain autoimmune diseases) that may increase the risk of
bleeding or thrombosis

- History of clinically significant hypersensitivity reaction associated with monoclonal
antibody therapies or components of the emicizumab injection

- Known human immunodeficiency virus (HIV) infection with CD4 count <200 cells/µL within
6 months prior to screening

- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or
planned use during the study, with the exception of antiretroviral therapy

- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could
interfere with the conduct of the study or that would, in the opinion of the
investigator or Sponsor, preclude the patient's safe participation in and completion
of the study or interpretation of the study results

- Receipt of: Emicizumab in a prior investigational study; An investigational drug to
treat or reduce the risk of hemophilic bleeds within five half-lives of last drug
administration; A non-hemophilia-related investigational drug within last 30 days or
five half-lives, whichever is shorter; or, Any concurrent investigational drug.

- Pregnancy or lactation, or intent to become pregnant during the study

- Positive serum pregnancy test result within 7 days prior to initiation of emicizumab
(females only)