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

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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Oktokog alpha

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.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 4

Contacts and Locations

Study Locations

• Sweden
  • Malmo, Sweden, SE-20502
    • Malmö Centre for Thrombosis and Haemostasis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 80 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Oktokog alpha (Advate); Personalized treatment according to individual PK using intravenous injection of oktokog alpha with dose and dose interval according to MyPKFIT and phenotypic evaluation.	Drug: Oktokog alpha; adjusting dose and dose interval according to MyPKFIT

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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	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	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	3 years

Collaborators and Investigators

• Sponsor: Lund University

Study record dates

Study Major Dates

• Study Start: December 1, 2016
• Primary Completion (Actual): April 9, 2021
• Study Completion (Actual): April 9, 2021

Study Registration Dates

• First Submitted: December 13, 2016
• First Submitted That Met QC Criteria: April 11, 2019
• First Posted (Actual): April 16, 2019

Study Record Updates

• Last Update Posted (Actual): April 13, 2021
• Last Update Submitted That Met QC Criteria: April 12, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: 20160920