Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor
Conditions
Brief summary
To evaluate Safety and efficacy and pharmacokinetics, FVIII Inhibitor titers of Hemlibra subcutaneous injection (SC inj.) in Korean Hemophilia A patients with/without FVIII Inhibitors.
Detailed description
Target subject : 1. Patients with Hemophilia A with FVIII inhibitors 2. Patients with severe Hemophilia A without FVIII inhibitors.
Interventions
DRUGEmicizumab subcutaneous injection
Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.
Sponsors
JW Pharmaceutical
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Patients with Hemophilia A (congenital factor VIII deficiency) who will receive Hemlibra SC inj. according to medical decision by investigator 2. Signed informed consent form
Exclusion criteria
1. Subjects who are hypersensitive to Emicizumab 2. Subjects who are hypersensitive to mouse or hamster protein 3. Subjects with myocardial infarction or the history who are prohibited from administering L-Arginine containing drug 4. Subjects who participated in other clinical trials within a month before enrollment (or the first administration of Hemlibra SC inj.) (Unable to participate in other clinical trials related to Hemophilia A treatment even after enrollment) 5. Subjects who the investigator deems inappropriate for the study. 6. Moderate/Mild for Hemophilia A patients without FVIII inhibitors
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events | for 24weeks | AEs will be described for AEs and Adverse Drug Reactions (ADRs), Adverse Events of Special Interest (AESIs), other important AEs. Severity, outcome, causal relationship with study drug, etc. of AEs will be analyzed. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Bleeding episode Rate (ABR) | for 24weeks | The number of bleeds will be also annualized for each patient using the following formula |
Countries
South Korea
Contacts
Primary ContactChuhl Joo Lyu, Prof
Backup ContactYoungShil Park, Prof.
Outcome results
None listed