Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

Lund University14 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

Oktokog alphaDRUG

adjusting dose and dose interval according to MyPKFIT

Eligibility

Sex: MALEMin age: 1 YearMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (1)

Malmö Centre for Thrombosis and Haemostasis

Malmö, Sweden

Outcomes

Primary Outcomes

PK measurement to individualize replacement therapy

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.

Time frame: 3 years

PK measurement to influence FVIII consumption

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

Time frame: 3 years

Specific pharmacokinetic parameters to analyze

Biological half-life of infused FVIII product will be measured in hours. Area under the curve (AUC)will be given in IUxh/dL

Time frame: 3 years

Secondary Outcomes

Signs of hemophilic arthropathy

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.

Time frame: 3 years

Data from ClinicalTrials.gov

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