NCT03533504

Brief Summary

Using anonymized patient data collected as part of the WAPPS-Hemo project to explore the sources of variability in individual pharmacokinetics (PK); use the sources of variability to improve the performance of the WAPPS-Hemo models through the addition of the predictors of PK variability as covariates.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
1,000

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started May 2018

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 9, 2018

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

May 10, 2018

Completed
13 days until next milestone

First Posted

Study publicly available on registry

May 23, 2018

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2024

Completed
Last Updated

March 15, 2023

Status Verified

March 1, 2023

Enrollment Period

6.1 years

First QC Date

May 10, 2018

Last Update Submit

March 14, 2023

Conditions

Hemophilia AHemophilia B

Keywords

pharmacokineticshemophiliafactor VIIfactor IXpopulation pharmacokinetics

Outcome Measures

Primary Outcomes (1)

  • Sources of variability in individual pharmacokinetics for factor VII and IX concentrates

    Multivariate hierarchical analysis will be performed on various factors (age, sex, weight, height, dose and type of factor administered (as total and IU/kg), blood group, baseline factor level, positive history of inhibitors, hematocrit, hemoglobin, and serum creatinine, laboratory methods used to measure factor VIII and factor IX) to explore sources of variability in patient outcome. Significant predictors will be used as covariates to improve the performance of WAPPS-Hemo models.

    2 years

Study Arms (1)

Variability in individual PK

Patients with hemophilia A or B, of any severity, who are registered on the web-accessible population pharmacokinetics- hemophilia (WAPPS-Hemo) database, and for whom infusion and/or PK data is available.

Other: Variability in individual PK

Interventions

Variability in individual PKOTHER

Multivariate hierarchical analysis will be performed on various factors (age, sex, weight, height, dose and type of factor administered (as total and IU/kg), blood group, baseline factor level, positive history of inhibitors, hematocrit, hemoglobin, and serum creatinine, laboratory methods used to measure factor VIII and factor IX) to explore sources of variability in patient outcome.

Variability in individual PK

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participates are individuals with hemophilia A or B, any severity, who have agreed to input of their data in the WAPPS-Hemo database, and who have sufficient infusion/PK data.

You may qualify if:

  • individuals with hemophilia A or B
  • infusion/PK data is available on the WAPPS-Hemo database
  • given informed consent to data input on the WAPPS-Hemo database

You may not qualify if:

  • \- individuals currently undergoing immune tolerance induction

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

McMaster University

Hamilton, Ontario, L8S4B2, Canada

Location

Related Publications (2)

  • Hajducek DM, Chelle P, Hermans C, Iorio A, McEneny-King A, Yu J, Edginton A. Development and evaluation of the population pharmacokinetic models for FVIII and FIX concentrates of the WAPPS-Hemo project. Haemophilia. 2020 May;26(3):384-400. doi: 10.1111/hae.13977. Epub 2020 Apr 13.

    PMID: 32281726RESULT

  • Hajducek DM, Sinkovic O, Chelle P, Iorio A, Edginton A. A Global Cross-Sectional Database Study of Low Dose FVIII SHL Prophylaxis in Haemophilia A. Haemophilia. 2025 Jul;31(4):646-656. doi: 10.1111/hae.70061. Epub 2025 May 10.

    PMID: 40347119DERIVED

Related Links

MeSH Terms

Conditions

Hemophilia AHemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Alfonso Iorio

    McMaster University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 10, 2018

First Posted

May 23, 2018

Study Start

May 9, 2018

Primary Completion

June 30, 2024

Study Completion

June 30, 2024

Last Updated

March 15, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

CA(1)