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Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A Using PK Measurement (myPKFiT) (myPKFiT)

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ClinicalTrials.gov Identifier: NCT03915080
Recruitment Status : Recruiting
First Posted : April 15, 2019
Last Update Posted : April 15, 2019
Sponsor:
Information provided by (Responsible Party):
Lund University

Brief Summary:
MyPKFiT is a web-based application recently developed by Baxalta for the use in patients treated with Advate. MyPkFit has its basis in Bayesian forecasting, which allows estimation of individual PK parameters by a sparse sampling schedule, where only 2-3 samples are taken between 4 and 48 hours post infusion. With myPKFiT, it will, therefore, be possible to define an individual PK curve for each patient based on just a few sampling points and hence, taking the bleeding phenotype and the life style into account, potentially adjust the prophylactic treatment accordingly to optimize cost-effectiveness.

Condition or disease Intervention/treatment Phase
Hemophilia A Drug: Advate Phase 4

Detailed Description:
As detailed in brief summary patients will, after accurate information verbally and written according to ethics approval, report bleeding events during previous 6 months. Blood samples are taken, FVIII analyzed (Advate patients) and the individual PK curve determined using MyPKFit. The curve will be discussed together with the patient and levels related to bleed events determined. If needed dose adjustment is done based on the curve. After a further 6 month follow-up bleeds will again be evaluated as well as factor consumption. In this way a better personalized treatment based on PK and clinical phenotype will hopefully be achieved.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A Using PK Measurement (myPKFiT)
Study Start Date : December 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 18, 2019


Arm Intervention/treatment
Advate
Personalized treatment according to individual PK
Drug: Advate



Primary Outcome Measures :
  1. PK measurement to individualize replacement therapy [ Time Frame: 3 years ]
    To evaluate whether PK measurement by myPKFiT and the calculation of an individualized PK profile may allow the treating physician to personalize and optimize the treatment of patients with haemophilia A to minimize the number of bleeds in a cost-effective way without diminishing compliance.

  2. PK measurement to influence FVIII consumption [ Time Frame: 3 years ]
    2. To calculate whether optimization of treatment by use of MyPKFiT, as described in primary objective 1, result in change of total FVIII consumption and extra doses given before and after visit 1. Measurements are T1/2, area under the curve

  3. Specific pharmacokinetic parameters to analyze [ Time Frame: 3 years ]
    Biological half-life of infused FVIII product will be measured in hours. Area under the curve (AUC)will be given in IUxh/dL


Secondary Outcome Measures :
  1. Signs of hemophilic arthropathy [ Time Frame: 3 years ]
    1. To identify and characterize and sign of arthropathy by ultrasound sound evaluation of the synovium, cartilage and bone according to the HEAD-US score of ankle, knee and elbows visit 1 and 2 and correlate this to the treatment provided.



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Ages Eligible for Study:   1 Year to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children and adults with severe hemophilia A (FVIII:C <1 %), being treated with Advate for more than 50 exposure days (EDs

Exclusion Criteria:

  • Current evidence of inhibitor as measured by the Nijmegen-modified Bethesda assay
  • Use of another investigational FVIII product in the previous month

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03915080


Contacts
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Contact: Erik Berntorp, Professor 0705752496 ext +46 erik.berntorp@med.lu.se
Contact: Alexandros Arvanitakis, MD

Locations
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Sweden
Malmö Centre for Thrombosis and Haemostasis Recruiting
Malmo, Sweden, SE-20502
Contact: Alexandros Arvanitakis, MD    +46 46 17 27 01    alexandros.arvanitakis@med.lu.se   
Contact: Jan Astermark, Professor       jan.astermark@med.lu.se   
Sub-Investigator: Alexandros Arvanitakis, MD         
Principal Investigator: Erik Berntorp, Professor         
Principal Investigator: Jan Astermark, Professor         
Sponsors and Collaborators
Lund University

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Responsible Party: Lund University
ClinicalTrials.gov Identifier: NCT03915080     History of Changes
Other Study ID Numbers: 20160920
First Posted: April 15, 2019    Key Record Dates
Last Update Posted: April 15, 2019
Last Verified: January 2019

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants