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

NCT03006965 — Completed, 50 participants, Spanish Society of Thrombosis and Haemostasis

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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

Registry	NCT03006965
Status	Completed
Study type	Observational
Enrollment	50 (Actual)
Sponsor	Spanish Society of Thrombosis and Haemostasis (Other); collaborators: Takeda, Instituto de Investigación Hospital Universitario La Paz
Conditions	Hemophilia A, Hemophilia, Factor VIII Deficiency
Interventions	octocog alfa, rurioctocog alfa pegol (Drug)
Dates	start 2016-11-11, primary completion 2022-12-22, completion 2022-12-22, first posted 2016-12-30, last updated 2023-06-08
Locations	14 sites in Spain

-Summary

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

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+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.

+Arms and interventions

Arm	Type	Interventions	Description
Hemophilia A patients		Drug: octocog alfa; Drug: 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.

octocog alfa (Drug), also known as Advate®, recombinant intravenous factor VIII: Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
rurioctocog alfa pegol (Drug), also known as recombinant intravenous factor VIII: Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software

+Outcome measures

Primary outcomes

Pharmacokinetic profile: FVIII half-life measured with MyPKFiT [3-4 hours postdose +/- 30 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT [24-32 hours postdose +/- 60 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT [48 hours postdose +/- 60 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: FVIII half-life measured with MyPKFiT [72 hours postdose]: Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT [3-4 hours postdose +/- 30 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT [24-32 hours postdose +/- 60 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: Clearance measured with MyPKFiT [48 hours postdose +/- 60 minutes]: 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 [3-4 hours postdose +/- 30 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT [24-32 hours postdose +/- 60 minutes]: Change from Baseline to 12 months
Pharmacokinetic profile: Volume in steady state measured with MyPKFiT [48 hours postdose +/- 60 minutes]: 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 outcomes

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

+Eligibility

Sex: Male, ages: 1 Year to 65 Years

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.

+Locations (14)

Facility	City	Country
Complexo Hospitalario Universitario A Coruña	A Coruña, A Coruña	Spain
Hospital Universitario Son Espases	Palma, Balearic Islands	Spain
Hospital Sant Joan de Deu	Esplugues de Llobregat, Barcelona	Spain
Hospital Universitario Virgen de la Arrixaca	El Palmar, Murcia	Spain
Hospital Virgen del Camino	Pamplona, Navarre	Spain
Hospital Xeral de Vigo	Vigo, Pontevedra	Spain
Hospital Universitario Central de Asturias	Oviedo, Principality of Asturias	Spain
Hospital Universitario Cruces	Barakaldo, Vizcaya	Spain
Hospital Vall D'Hebron	Barcelona	Spain
Hospital Universitario La Paz	Madrid	Spain
Hospital Regional Universitario de Málaga	Málaga	Spain
Hospital Materno-infantil de Málaga	Málaga	Spain
Hospital Universitario Virgen del Rocío	Seville	Spain
Hospital Universitari i Politècnic La Fe	Valencia	Spain

+Investigators

M Teresa Álvarez Román, MD,PhD, Principal investigator, Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid

My Notes

Public

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