Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
- Conditions
- Hemophilia AFactor VIII DeficiencyHemophilia
- Interventions
- Registration Number
- NCT03006965
- Lead Sponsor
- Spanish Society of Thrombosis and Haemostasis
- Brief Summary
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 50
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Hemophilia A patients octocog alfa 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. Hemophilia A patients rurioctocog alfa pegol 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.
- Primary Outcome Measures
Name Time Method Pharmacokinetic profile: FVIII half-life measured with MyPKFiT 72 hours postdose Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT 72 hours postdose Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT 72 hours postdose Change from Baseline to 12 months
- Secondary Outcome Measures
Name Time Method Hemorrhagic risk of physical activity month 12 Measured with Broderick scale
Self-adherence to treatment evaluation month 12 Measured by VERITAS- PRO scale
Number of total bleeding episodes in a year month 12 Measured by annual bleeding record
Annual Joint Bleeding Record month 12 Hemophilia Joint Health Score
Joint condition month 12 Hemophilia Joint Health Score
Adverse Events month 12 Collect and identify adverse events
Trial Locations
- Locations (14)
Hospital Universitario Virgen de la Arrixaca
🇪🇸El Palmar, Murcia, Spain
Hospital Virgen del Camino
🇪🇸Pamplona, Navarra, Spain
Hospital Xeral de Vigo
🇪🇸Vigo, Pontevedra, Spain
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain
Hospital Universitario Son Espases
🇪🇸Palma, Islas Baleares, Spain
Complexo Hospitalario Universitario A Coruña
🇪🇸A Coruna, A Coruña, Spain
Hospital Universitario Cruces
🇪🇸Barakaldo, Vizcaya, Spain
Hospital Regional Universitario de Málaga
🇪🇸Malaga, Spain
Hospital Materno-infantil de Málaga
🇪🇸Malaga, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Hospital Vall D'Hebron
🇪🇸Barcelona, Spain
Hospital Universitari i Politècnic La Fe
🇪🇸Valencia, Spain
Hospital Universitario Central de Asturias
🇪🇸Oviedo, Asturias, Spain
Hospital Sant Joan de Deu
🇪🇸Esplugues de Llobregat, Barcelona, Spain