An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A

An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A - An Observational Study to Investigate the Effectiveness of PK Guided Personalized Recombinant (r)FVIII Treatment in Patients with Hemophilia A

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000044800

Enrollment

Registered

2021-07-09

Start date

2021-08-20

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Interventions

None listed

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Male patients with congenital severe/moderate hemophilia A (severe= FVIII:C <1% at baseline, moderate*= FVIII: 1% <= C < 5% at baseline) * : Patients receiving regular replacement therapy 2. Patients receiving or who had been treated with rurioctocog alfa and rurioctocog alfa pegol 3. Patients undergoing PK-guided personalized therapy (rFVIII replacement therapy) with myPKFiT 4. Age >=12 years at the time of initiation of myPKFiT (if on rurioctocog alfa pegol treatment)

Exclusion criteria

Exclusion criteria: 1. Patients using PK guided tools that are unapproved in Japan 2. Patients on FVIII inhibitors

Design outcomes

Primary

Measure	Time frame
Primary endpoint Annualized bleeding rate (ABR)	—

Secondary

Measure	Time frame
<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	—

Measure

Time frame

<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

—

Countries

Japan

Contacts

Public ContactYasuo Miyaguchi

Takeda Pharmaceutical Company Limited. Japan Medical Office

yasuo.miyaguchi@takeda.com070-4921-7966

Outcome results