Hemophilia
Conditions
Interventions
test group:emicizumab
Control group:FVIII prophylaxis
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Children with moderate or severe hemophilia A; 2. Aged < 12 years old; 3. The details of FVIII treatment and the number of bleeding events have been recorded for at least the last 24 weeks; 4. Inhibitor-negative medical history.
Exclusion criteria
Exclusion criteria: 1. There is a genetic bleeding disorder other than hemophilia A; 2. Has thrombotic disease or a family history of related diseases; 3. Previous allergy to monoclonal antibody therapy, emicerizumab, or allergic disease; 4. Liver function: total bilirubin > 1.5 times the upper limit of normal (ULN)) and AST and/or ALT > 3 times the upper limit of normal ULN; 5. Renal function meets the requirements: serum creatinine > 2.5 times ULN, and creatinine clearance calculated by Cockcroft-Gault formula < 30 mL/min.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Joint Ultrasound/MRI; | — |
Countries
China
Contacts
Public ContactWu Runhui
Beijing Children's Hospital, Capital Medical University
Outcome results
None listed