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

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ClinicalTrials.gov Identifier: NCT02750085
Recruitment Status : Enrolling by invitation
First Posted : April 25, 2016
Last Update Posted : October 30, 2018
Sponsor:
Collaborators:
St. Paul's Hospital
Montreal Children's Hospital of the MUHC
Queen's University
University Hospital, Motol
University Hospital Brno
Royal Children's Hospital
Sydney Children's Hospitals Network
Royal Prince Alfred Hospital, Sydney, Australia
Information provided by (Responsible Party):
Victor Blanchette, The Hospital for Sick Children

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.

Condition or disease
Hemophilia A

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia
Actual Study Start Date : April 2016
Estimated Primary Completion Date : April 2019
Estimated Study Completion Date : April 2019


Group/Cohort
2-point and 6-point PK sampling



Primary Outcome Measures :
  1. Terminal half-life (t1/2) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  2. area under the plasma concentration versus time curve (AUC) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  3. Area under the moment curve (AUMC) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  4. In vivo recovery (IVR) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  5. Maximum concentration (Cmax) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  6. Clearance (Cl) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  7. Volume of distribution at steady state (Vss) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  8. Mean residence time (MRT) [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol

  9. Time to factor VIII concentration of 1% over baseline [ Time Frame: 2 years ]
    2-point PK sampling protocol against 6-point PK sampling protocol


Biospecimen Retention:   Samples Without DNA
Plasma collected from PK blood sampling


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with severe Hemophilia A
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02750085


Sponsors and Collaborators
Victor Blanchette
St. Paul's Hospital
Montreal Children's Hospital of the MUHC
Queen's University
University Hospital, Motol
University Hospital Brno
Royal Children's Hospital
Sydney Children's Hospitals Network
Royal Prince Alfred Hospital, Sydney, Australia
Investigators
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Principal Investigator: Victor S Blanchette, MD The Hospital for Sick Children

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Responsible Party: Victor Blanchette, Medical Director, Pediatric Thrombosis and Hemostasis Program, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT02750085     History of Changes
Other Study ID Numbers: myPKFiT™ Study
First Posted: April 25, 2016    Key Record Dates
Last Update Posted: October 30, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Victor Blanchette, The Hospital for Sick Children:
Hemophilia A
pharmacokinetics
myPKFiT

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants