Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A

NCT03915080 | Completed Phase 4

• 1 other identifier: 20160920
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• Signs of hemophilic arthropathy

  3 years

Study Arms (1)

Oktokog alpha (Advate)

OTHER

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

Interventions

Oktokog alpha DRUG

adjusting dose and dose interval according to MyPKFIT

Oktokog alpha (Advate)

Eligibility Criteria

• Age: 1 Year - 80 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Children and adults with severe hemophilia A (FVIII:C \<1 %), being treated with Advate for more than 50 exposure days (EDs

You may not qualify if:

• Current evidence of inhibitor as measured by the Nijmegen-modified Bethesda assay
• Use of another investigational FVIII product in the previous month

Sponsors & Collaborators

• Lund University: lead

Study Sites (1)

Malmö Centre for Thrombosis and Haemostasis

Malmo, SE-20502, Sweden

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2016
• First Posted: April 16, 2019
• Study Start: December 1, 2016
• Primary Completion: April 9, 2021
• Study Completion: April 9, 2021
• Last Updated: April 13, 2021

Record last verified: 2021-04

Locations

SE (1)