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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03006965
• Other study ID #: PK HemoA-SP
• Secondary ID: STH-OCT-2016-01
• Status: Completed
• Start date: November 11, 2016
• Est. completion date: December 22, 2022

Study information

• Verified date: June 2023
• Source: Spanish Society of Thrombosis and Haemostasis
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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 information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: December 22, 2022
• Est. primary completion date: December 22, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 1 Year to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Factor VIII Deficiency
• Hemophilia
• Hemophilia A

Intervention

Drug:
• octocog alfa
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
• rurioctocog alfa pegol
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software

Locations

Country	Name	City	State
Spain	Complexo Hospitalario Universitario A Coruña	A Coruna	A Coruña
Spain	Hospital Universitario Cruces	Barakaldo	Vizcaya
Spain	Hospital Vall D'Hebron	Barcelona
Spain	Hospital Universitario Virgen de la Arrixaca	El Palmar	Murcia
Spain	Hospital Sant Joan de Deu	Esplugues de Llobregat	Barcelona
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Materno-infantil de Málaga	Malaga
Spain	Hospital Regional Universitario de Málaga	Malaga
Spain	Hospital Universitario Central de Asturias	Oviedo	Asturias
Spain	Hospital Universitario Son Espases	Palma	Islas Baleares
Spain	Hospital Virgen del Camino	Pamplona	Navarra
Spain	Hospital Universitario Virgen del Rocío	Sevilla
Spain	Hospital Universitari i Politècnic La Fe	Valencia
Spain	Hospital Xeral de Vigo	Vigo	Pontevedra

Sponsors (3)

Lead Sponsor	Collaborator
Spanish Society of Thrombosis and Haemostasis	Instituto de Investigación Hospital Universitario La Paz, Takeda

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pharmacokinetic profile: FVIII half-life measured with MyPKFiT	Change from Baseline to 12 months	3-4 hours postdose +/- 30 minutes
Primary	Pharmacokinetic profile: FVIII half-life measured with MyPKFiT	Change from Baseline to 12 months	24-32 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: FVIII half-life measured with MyPKFiT	Change from Baseline to 12 months	48 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: FVIII half-life measured with MyPKFiT	Change from Baseline to 12 months	72 hours postdose
Primary	Pharmacokinetic profile: Clearance measured with MyPKFiT	Change from Baseline to 12 months	3-4 hours postdose +/- 30 minutes
Primary	Pharmacokinetic profile: Clearance measured with MyPKFiT	Change from Baseline to 12 months	24-32 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: Clearance measured with MyPKFiT	Change from Baseline to 12 months	48 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: Clearance measured with MyPKFiT	Change from Baseline to 12 months	72 hours postdose
Primary	Pharmacokinetic profile: Volume in steady state measured with MyPKFiT	Change from Baseline to 12 months	3-4 hours postdose +/- 30 minutes
Primary	Pharmacokinetic profile: Volume in steady state measured with MyPKFiT	Change from Baseline to 12 months	24-32 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: Volume in steady state measured with MyPKFiT	Change from Baseline to 12 months	48 hours postdose +/- 60 minutes
Primary	Pharmacokinetic profile: Volume in steady state measured with MyPKFiT	Change from Baseline to 12 months	72 hours postdose
Secondary	Hemorrhagic risk of physical activity	Measured with Broderick scale	baseline
Secondary	Hemorrhagic risk of physical activity	Measured with Broderick scale	month 12
Secondary	Joint condition	Hemophilia Joint Health Score	baseline
Secondary	Joint condition	Hemophilia Joint Health Score	month 12
Secondary	Number of total bleeding episodes in a year	Measured by annual bleeding record	baseline
Secondary	Number of total bleeding episodes in a year	Measured by annual bleeding record	month 12
Secondary	Annual Joint Bleeding Record	Hemophilia Joint Health Score	baseline
Secondary	Annual Joint Bleeding Record	Hemophilia Joint Health Score	month 12
Secondary	Self-adherence to treatment evaluation	Measured by VERITAS- PRO scale	month 12
Secondary	Adverse Events	Collect and identify adverse events	baseline
Secondary	Adverse Events	Collect and identify adverse events	month 12