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

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

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

Study Overview

• Status: Completed
• Conditions: Hemophilia A; Hemophilia; Factor VIII Deficiency
• Intervention / Treatment: Drug: octocog alfa; Drug: rurioctocog alfa pegol

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.

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

• Study Contact: Name: M Teresa Álvarez Román, MD,PhD; Phone Number: +34 913582211; Email: talvarezroman@gmail.com
• Study Contact Backup: Name: Sara García Barcenilla; Phone Number: +34 913582211; Email: ehemostasia@gmail.com

Study Locations

• Spain
  • Barcelona, Spain, 08035
    • Hospital Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Malaga, Spain, 29010
    • Hospital Regional Universitario de Malaga
  • Malaga, Spain, 29011
    • Hospital Materno-Infantil de Malaga
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen Del Rocio
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe
  • A Coruña
    • A Coruna, A Coruña, Spain, 15006
      • Complexo Hospitalario Universitario A Coruña
  • Asturias
    • Oviedo, Asturias, Spain, 33011
      • Hospital Universitario Central de Asturias
  • Barcelona
    • Esplugues de Llobregat, Barcelona, Spain, 08950
      • Hospital Sant Joan de Déu
  • Islas Baleares
    • Palma, Islas Baleares, Spain, 07120
      • Hospital Universitario Son Espases
  • Murcia
    • El Palmar, Murcia, Spain, 30120
      • Hospital Universitario Virgen de la Arrixaca
  • Navarra
    • Pamplona, Navarra, Spain, 31008
      • Hospital Virgen del Camino
  • Pontevedra
    • Vigo, Pontevedra, Spain, 36212
      • Hospital Xeral de Vigo
  • Vizcaya
    • Barakaldo, Vizcaya, Spain, 48903
      • Hospital Universitario Cruces

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 months to 63 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with hemophilia A on prophylactic treatment with Advate® or Adynovi®

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Hemophilia A patients; 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; Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software; Other Names: Advate®; recombinant intravenous factor VIII; Drug: rurioctocog alfa pegol; Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software; Other Names: recombinant intravenous factor VIII

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Spanish Society of Thrombosis and Haemostasis
• Collaborators: Takeda; Instituto de Investigación Hospital Universitario La Paz
• Investigators: Principal Investigator: M Teresa Álvarez Román, MD,PhD, Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid

Study record dates

Study Major Dates

• Study Start (Actual): November 11, 2016
• Primary Completion (Actual): December 22, 2022
• Study Completion (Actual): December 22, 2022

Study Registration Dates

• First Submitted: December 22, 2016
• First Submitted That Met QC Criteria: December 29, 2016
• First Posted (Estimated): December 30, 2016

Study Record Updates

• Last Update Posted (Actual): June 8, 2023
• Last Update Submitted That Met QC Criteria: June 7, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Factor VIII; Hemophilia; Hemophilia A; Hemarthrosis; Advate; Antithrombotic factor; Pharmacokinetic dosing; myPKFiT
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII; BAX 855
• Other Study ID Numbers: PK HemoA-SP; STH-OCT-2016-01 (Other Identifier: AEMPS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No