Hemophilia A
Conditions
Sponsors
CHUGAI PHARMACEUTICAL CO., LTD
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Children 3 kg at the time of informed consent 3) Diagnosis of severe congenital hemophilia A (i.e., endogenous FVIII activity level = 2 years of age: Documentation of bleeds and treatment with coagulation factor in the last 24 weeks prior to enrollment
Exclusion criteria
Exclusion criteria: 1) Inherited or acquired bleeding disorder other than hemophilia A 2) Ongoing immune tolerance induction (ITI) therapy 3) Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease 4) Other diseases (i.e., certain autoimmune disease [e.g., systemic lupus erythematosus, antiphospholipid antibody syndrome], cardiovascular disease [e.g., fallot's tetralogy, tricuspid atresia]) that may increase risk of bleeding or thrombosis 5) Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA [e.g., thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome]), in the investigator's judgment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Observation | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy pharmacokinetics safety Observation, Laboratory tests | — |
Countries
Japan
Outcome results
None listed