Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia

Brief Title

Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia

Official Title

Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia

Brief Summary

      This is an investigator-initiated, industry-funded, multi-centre, international study that
      will be carried out prospectively at hemophilia treatment centres across Canada, the Czech
      Republic and Australia with SickKids as the coordinating site. The study will use a central
      laboratory not directly affiliated with any of the participating sites. Enrollment target is
      50 participants, both adult and pediatric with severe hemophilia A receiving Advate, who will
      each complete a 2-point and 6-point pharmacokinetic (PK) sampling. The main aim is to compare
      the results of a 2 sample PK using clinically practical time points and myPKFiT™ (a
      web-based, population PK Bayesian tool) to a 6 sample population PK to determine whether the
      results obtained are in good agreement.

Study Type

Observational

Primary Outcome

Terminal half-life (t1/2)

Condition

Hemophilia A

Study Arms / Comparison Groups

 2-point and 6-point PK sampling

Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Estimated Enrollment

Start Date

April 2016

Completion Date

December 2020

Primary Completion Date

January 2019

Eligibility Criteria

        Inclusion Criteria:

          -  Confirmed diagnosis of Hemophilia A;

          -  Severe disease (FVIII <2%);

          -  Receiving ADVATE for prevention of bleeding (prophylaxis) or receiving ADVATE on
             demand and a candidate for prophylaxis;

          -  Body weight ≤120 kg; and ≥12kg;

        Exclusion Criteria:

          -  FVIII inhibitor positive (level of ≥0.6 Bethesda Units [BU] per mL using the Nijmegen
             modification of the Bethesda assay). Inhibitor status to be documented as negative
             prior to study enrollment according to the two most recent, consecutive inhibitor
             assays on record. If patients have < 50 exposure days, an assay will be completed
             centrally within a reasonable timeframe (approximately 8 weeks suggested) to make sure
             that they are negative.

          -  Body weight >120 kg or <12kg;

          -  Human immunodeficiency virus (HIV) positivity with cluster of differentiation 4 (CD4)
             count < 200 / microliter;

          -  Significant hepatic dysfunction, alanine aminotransferase (ALT) or aspartate
             aminotransferase (AST) >5 times upper limit of normal

          -  History of recent events that might affect FVIII half-life (e.g., infection, surgery
             or an invasive procedure) within 2 weeks of blood sampling.

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Victor S Blanchette, MD, ,

Administrative Informations

NCT ID

NCT02750085

Organization ID

myPKFiT™ Study

Responsible Party

Sponsor-Investigator

Study Sponsor

Victor Blanchette

Collaborators

 St. Paul's Hospital

Study Sponsor

Victor S Blanchette, MD, Principal Investigator, The Hospital for Sick Children

Verification Date

May 2020