An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A
Not Applicable
- Conditions
- Hemophilia A
- Registration Number
- JPRN-UMIN000044800
- Lead Sponsor
- Takeda Pharmaceutical Company Limited.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- Male
- Target Recruitment
- 30
Inclusion Criteria
Not provided
Exclusion Criteria
1. Patients using PK guided tools that are unapproved in Japan 2. Patients on FVIII inhibitors
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Primary endpoint Annualized bleeding rate (ABR)
- Secondary Outcome Measures
Name Time Method <Secondary endpoints> 1.ABR by each of the following details of bleeding (traumatic hemorrhage, spontaneous hemorrhage, joint hemorrhage, non-joint hemorrhage) 2.Consumption of rFVIII products 3.Amount of physical activity 4.QOL 5.Satisfaction with the myPKFiT mobile application (only for users) <Exploratory evaluations> 1.Target and observed trough (only if evaluable) 2.Number of rFVIII administrations per week