Hemophilia A with inhibitors
Conditions
Sponsors
Chugai Pharmaceutical Co., Ltd.
F. Hoffmann-La Roche Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)Aged 12 years or older at the time of informed consent 2)Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e., >= 5 Bethesda Units) 3)Documentation of treatment with episodic or prophylactic bypassing agents for at least the last 24 weeks 4)6 (if on an episodic bypassing agent regimen) or >= 2 (if on a prophylactic bypassing agent regimen) bleeds in the last 24 weeks
Exclusion criteria
Exclusion criteria: 1) Inherited or acquired bleeding disorder other than hemophilia A 2) Ongoing (or plan to receive during the study) immune tolerance induction therapy or prophylaxis with FVIII 3) Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or signs of thromboembolic disease 4) Previous or concurrent autoimmune or connective tissue disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy primary outcome: To evaluate the efficacy of prophylactic emicizumab compared with no prophylaxis in hemophilia A patients with inhibitors on the basis of number of bleeds over time. primary outcome method: Observation | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy pharmacokinetics secondary outcome: To evaluate the efficacy, safety and pharmacokinetics of prophylactic emicizumab secondary outcome method:Observation, Laboratory tests | — |
Countries
14 countries including Japan, Japan
Outcome results
None listed