Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

Hemophilia A Clinical Trial

— myPKFiT  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03915080
• Other study ID #: 20160920
• Status: Completed
• Phase: Phase 4
• Start date: December 2016
• Est. completion date: April 9, 2021

Study information

• Verified date: April 2021
• Source: Lund University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: April 9, 2021
• Est. primary completion date: April 9, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 1 Year to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Oktokog alpha
adjusting dose and dose interval according to MyPKFIT

Locations

Country	Name	City	State
Sweden	Malmö Centre for Thrombosis and Haemostasis	Malmo

Sponsors (1)

Lead Sponsor	Collaborator
Lund University

Country where clinical trial is conducted

Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	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