Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®

Study Overview
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Brief Summary
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The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®

Detailed Description
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This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products, with myPKFiT®. This patients will be followed during the period of 12 months, and the pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence of factor VIII will be determined by the usual clinical methods: chromogenic method or coagulative method.

Official Title
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Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm

Conditions
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Hemophilia A, Hemophilia, Factor VIII Deficiency

Intervention / Treatment
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* Drug: octocog alfa
* Drug: rurioctocog alfa pegol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male patients. Age between 1 and 65 years old. Patients diagnosed with Hemophilia A who are on prophylactic treatment with Advate® or Adynovi® or adjusted with myPKFIT. Patients older than 18 who have signed the informed consent form. In the case of mature minors, in addition to the consent signed by the legal guardian, an assent of the minor must be obtain. In the case of patients legally incapable for giving their consent, their primary caregiver will consent as the patient's legal guardian. Exclusion Criteria: Withdrawal of informed consent. Patients with any medical or psychological condition that according to the researcher's criteria prevents them from following the usual clinical practice procedures. Patients with concomitant diagnosis of other haemostasis disorders. Patients being treated for induction of immunologic tolerance at the time of inclusion.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 65 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Hemophilia A patients<br>Group of patients in prophylactic treatment with Advate® (octocog alfa) or Adynovi® (rurioctocog alfa pegol), or patients using already myPKFit®. Patients will be given a dose of octocog alfa or rurioctocog alfa pegol according to usual clinical practice, and two blood samples will be taken in case of octocog alfa: one sample will be extracted 3-4h postdose (+/- 30 minutes), and the second sample will be extracted 24-32h postdose (+/- 60 minutes). In case of rurioctocog alfa pegol, the first sample is taken in the same conditions than octocog alfa, and the second sample will be extracted 48h postdose (+/- 120 minutes), and other sample post 72h(+/- 120 minutes) optional. | Drug: octocog alfa<br>* Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software<br>* Other names: recombinant intravenous factor VIII;Drug: rurioctocog alfa pegol<br>* Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software<br>* Other names: recombinant intravenous factor VIII;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetic profile: FVIII half-life measured with MyPKFiT | Change from Baseline to 12 months | 3-4 hours postdose +/- 30 minutes | 
| Pharmacokinetic profile: FVIII half-life measured with MyPKFiT | Change from Baseline to 12 months | 24-32 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: FVIII half-life measured with MyPKFiT | Change from Baseline to 12 months | 48 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: FVIII half-life measured with MyPKFiT | Change from Baseline to 12 months | 72 hours postdose | 
| Pharmacokinetic profile: Clearance measured with MyPKFiT | Change from Baseline to 12 months | 3-4 hours postdose +/- 30 minutes | 
| Pharmacokinetic profile: Clearance measured with MyPKFiT | Change from Baseline to 12 months | 24-32 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: Clearance measured with MyPKFiT | Change from Baseline to 12 months | 48 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: Clearance measured with MyPKFiT | Change from Baseline to 12 months | 72 hours postdose | 
| Pharmacokinetic profile: Volume in steady state measured with MyPKFiT | Change from Baseline to 12 months | 3-4 hours postdose +/- 30 minutes | 
| Pharmacokinetic profile: Volume in steady state measured with MyPKFiT | Change from Baseline to 12 months | 24-32 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: Volume in steady state measured with MyPKFiT | Change from Baseline to 12 months | 48 hours postdose +/- 60 minutes | 
| Pharmacokinetic profile: Volume in steady state measured with MyPKFiT | Change from Baseline to 12 months | 72 hours postdose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemorrhagic risk of physical activity | Measured with Broderick scale | baseline | 
| Hemorrhagic risk of physical activity | Measured with Broderick scale | month 12 | 
| Joint condition | Hemophilia Joint Health Score | baseline | 
| Joint condition | Hemophilia Joint Health Score | month 12 | 
| Number of total bleeding episodes in a year | Measured by annual bleeding record | baseline | 
| Number of total bleeding episodes in a year | Measured by annual bleeding record | month 12 | 
| Annual Joint Bleeding Record | Hemophilia Joint Health Score | baseline | 
| Annual Joint Bleeding Record | Hemophilia Joint Health Score | month 12 | 
| Self-adherence to treatment evaluation | Measured by VERITAS- PRO scale | month 12 | 
| Adverse Events | Collect and identify adverse events | baseline | 
| Adverse Events | Collect and identify adverse events | month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia, Hemophilia A, Factor VIII, Advate, Antithrombotic factor, Pharmacokinetic dosing, Hemarthrosis, myPKFiT