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NCT Number: NCT03915080

Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

1 year–80 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

Primary location

Malmö Centre for Thrombosis and Haemostasis

Malmö, SE-20502, Sweden

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Oktokog alpha

Drug

adjusting dose and dose interval according to MyPKFIT

Primary outcomes

  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

    • 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 outcomes

  1. Signs of hemophilic arthropathy

    Time frame: 3 years

    • 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.

Sponsors and collaborators

Lead sponsor

Lund University

Other

Registry information

Official study title

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

Acronym: myPKFiT

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Apr 16, 2019
Registry last updated
Apr 13, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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