Clinical trial NCT03006965: Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Hemophilia
Factor VIII Deficiency
Keywords: Hemophilia
Hemophilia A
Factor VIII
Advate
Antithrombotic factor
Pharmacokinetic dosing
Hemarthrosis
myPKFiT
Lead sponsor: Spanish Society of Thrombosis and Haemostasis
Sponsor class: OTHER
Collaborators: name: Takeda
name: Instituto de Investigación Hospital Universitario La Paz
First posted: 2016-12-30
Enrollment: 50
Sex: MALE
Minimum age (years): 1
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with hemophilia A on prophylactic treatment with Advate® or Adynovi®

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
timeFrame: 3-4 hours postdose +/- 30 minutes
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
timeFrame: 24-32 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
timeFrame: 48 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
timeFrame: 72 hours postdose
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
timeFrame: 3-4 hours postdose +/- 30 minutes
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
timeFrame: 24-32 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
timeFrame: 48 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
timeFrame: 72 hours postdose
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
timeFrame: 3-4 hours postdose +/- 30 minutes
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
timeFrame: 24-32 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
timeFrame: 48 hours postdose +/- 60 minutes
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
timeFrame: 72 hours postdose
Secondary outcomes:
measure: Hemorrhagic risk of physical activity
timeFrame: baseline
measure: Hemorrhagic risk of physical activity
timeFrame: month 12
measure: Joint condition
timeFrame: baseline
measure: Joint condition
timeFrame: month 12
measure: Number of total bleeding episodes in a year
timeFrame: baseline
measure: Number of total bleeding episodes in a year
timeFrame: month 12
measure: Annual Joint Bleeding Record
timeFrame: baseline
measure: Annual Joint Bleeding Record
timeFrame: month 12
measure: Self-adherence to treatment evaluation
timeFrame: month 12
measure: Adverse Events
timeFrame: baseline
measure: Adverse Events
timeFrame: month 12
Other outcomes:
Locations:
city: A Coruna
country: Spain
facility: Complexo Hospitalario Universitario A Coruña
state: A Coruña
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
state: Asturias
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
state: Barcelona
city: Palma
country: Spain
facility: Hospital Universitario Son Espases
state: Islas Baleares
city: El Palmar
country: Spain
facility: Hospital Universitario Virgen de la Arrixaca
state: Murcia
city: Pamplona
country: Spain
facility: Hospital Virgen del Camino
state: Navarra
city: Vigo
country: Spain
facility: Hospital Xeral de Vigo
state: Pontevedra
city: Barakaldo
country: Spain
facility: Hospital Universitario Cruces
state: Vizcaya
city: Barcelona
country: Spain
facility: Hospital Vall D'Hebron
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Málaga
city: Malaga
country: Spain
facility: Hospital Materno-infantil de Málaga
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío
city: Valencia
country: Spain
facility: Hospital Universitari i Politècnic La Fe
Study officials:
affiliation: Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid
name: M Teresa Álvarez Román, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References: