Bleeder's disease
Conditions
Interventions
Patients will receive treatment with emicizumab during this study. Emicizumab
is administered through the use of an injection just beneath the surface of the
skin. You will receive one dose of emici
Emicizumab
Hemophilia A
Sponsors
Roche Nederland B.V.
Eligibility
Age
12 Years to 64 Years
Inclusion criteria
Inclusion criteria: - As per the investigator's judgment, 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 - Body weight >
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of this study is to evaluate the overall safety and tolerability of prophylactic administration of emicizumab in patients with hemophilia A. The specific focus will be to characterize any thromboembolic events, microangiopathic hemolytic anemia, thrombotic microangiopathy (TMA) (e.g. hemolytic uremic syndrome), and hypersensitivity reactions that may occur in patients with hemophilia A treated with emicizumab. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objective of this study is to evaluate the efficacy of prophylactic administration of emicizumab. As part of this objective, the number of bleeds over time will be recorded for all of the enrolled patients. | — |
Countries
Australia, Belgium, Brazil, Bulgaria, Canada, Colombia, Denmark, Dominican Republic, Finland, Germany, Guatemala, Hungary, Israel, Italy, Mexico, Netherlands, Panama, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Spain, Sweden, Switzerland, United Kingdom
Outcome results
None listed