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

NCT03006965 | Completed

• 2 other identifiers: PK HemoA-SPSTH-OCT-2016-01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 14

Brief Summary

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

Conditions

Hemophilia A; Hemophilia; Factor VIII Deficiency

Keywords

Hemophilia; Hemophilia A; Factor VIII; Advate; Antithrombotic factor; Pharmacokinetic dosing; Hemarthrosis; myPKFiT

Outcome Measures

Primary Outcomes (12)

• 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 Outcomes (11)

• Hemorrhagic risk of physical activity

  baseline

• Hemorrhagic risk of physical activity

  month 12

• Joint condition

  baseline

• Joint condition

  month 12

• Number of total bleeding episodes in a year

  baseline

Study Arms (1)

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; Drug: rurioctocog alfa pegol

Interventions

octocog alfa DRUG

Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software

Also known as: Advate®, recombinant intravenous factor VIII

Hemophilia A patients

rurioctocog alfa pegol DRUG

Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software

Also known as: recombinant intravenous factor VIII

Hemophilia A patients

Eligibility Criteria

• Age: 1 Year - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Spanish Society of Thrombosis and Haemostasis: lead
• Takeda: collaborator
• Instituto de Investigación Hospital Universitario La Paz: collaborator

Study Sites (14)

Complexo Hospitalario Universitario A Coruña

A Coruña, A Coruña, 15006, Spain

Location

Hospital Universitario Son Espases

Palma, Balearic Islands, 07120, Spain

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Hospital Universitario Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

Hospital Virgen del Camino

Pamplona, Navarre, 31008, Spain

Location

Hospital Xeral de Vigo

Vigo, Pontevedra, 36212, Spain

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

Location

Hospital Universitario Cruces

Barakaldo, Vizcaya, 48903, Spain

Location

Hospital Vall D'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29010, Spain

Location

Hospital Materno-infantil de Málaga

Málaga, 29011, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

Location

Hospital Universitari i Politècnic La Fe

Valencia, 46026, Spain

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Whole blood samples collected in citrate plasma tubes to separate it and measure FVIII:chromogenic and FVIII:one stage

MeSH Terms

Conditions

Hemophilia A; Hemarthrosis

Interventions

F8 protein, human; Factor VIII; BAX 855

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Joint Diseases; Musculoskeletal Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• M Teresa Álvarez Román, MD,PhD

  Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2016
• First Posted: December 30, 2016
• Study Start: November 11, 2016
• Primary Completion: December 22, 2022
• Study Completion: December 22, 2022
• Last Updated: June 8, 2023

Record last verified: 2023-06

Locations

ES (14)