Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

• Status: Completed
• Trial end date: 2021-04-09
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Lund University
• Treatments: Factor VIII

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