Recruiting

WAPPS-Hemo

Sponsor:

McMaster University

Code:

NCT03615053

Conditions

Hemophilia A

Hemophilia B

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

Implementation of WAPPS-Hemo personalized dosing regimen

Study Details

Brief summary:

Performing an individual pharmacokinetic (PK) estimate is only the first step in implementing tailored prophylaxis, which requires using the PK profile information to design a personalized treatment regimen matching the treatment needs of individual patients. The overarching goal of WAPPS-Hemo is to provide an easy-to-use web application supporting all the steps needed to accomplish tailoring care of individual patients by matching their unique characteristics to the most appropriate treatment regimen, realizing the promise of personalized medicine.

This study will assess the impact of adopting population PK (popPK) based tailored prophylaxis in clinical practice, including proportion of patients eligible for tailoring, and encountered barriers. The impact on patient important outcomes and on societal outcomes, particularly financial impact, vs. current standardized regimens will be measured. It is hypothesized that WAPPS-Hemo, via estimation of precise individual PK profiles and by supporting the simulation of treatment regimens will:

1. improve or maintain patient important outcomes, while reducing wastage of factor concentrates; and
2. establish best practices and effective knowledge translation strategies for the implementation of personalized medicine.

Additionally, a solid base of data will be generated to model the bleeding risk of severe hemophilia A/B patients undergoing tailored prophylaxis which will enable evaluation of a combination of patient and treatment characteristics predictive of individual bleeding risk.

Conditions

Hemophilia A

Hemophilia B

Study ID

NCT03615053

Start date

Jul 24, 2019

Status verified date

Mar, 2023

Completion date

Sep 30, 2023

Anticipated

Primary completion date

Sep 30, 2022

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • individuals with severe congenital hemophilia A and B;
  • on continuous factor prophylaxis;
  • must be registered on CBDR (iCHIP in BC)

Exclusion Criteria:

  • a history of explicit and documented previous treatment tailoring based on pharmacokinetic profiling;
  • another congenital or acquired bleeding disorders other than Hemophilia A or B;
  • active inhibitors (> 5 Bethesda units) or currently undergoing immune tolerance induction.

Study Design

Enrollment

600 participants

Anticipated

Intervention Model

Single group

Primary purpose

Other

Interventions and Outcome Measures

Arms

other: Tailored Regimen

Implementation of WAPPS-Hemo personalized dosing regimen.

Interventions

Implementation of WAPPS-Hemo personalized dosing regimen

Twelve months after enrollment in the study, WAPPS-Hemo will be used to calculate each patients individual pharmacokinetic estimate. Once completed, the WAPPS-Hemo clinical calculator will be used to calculate the optimal regimen based on individual needs.

Primary outcome measure

  • Change in patient quality of life [ Time Frame: Completed every 3-6 months for duration of the study, from enrollment to study completion. ]
  • Annualized Bleeding Rate (ABR) pre and post-tailoring implementation [ Time Frame: Recorded throughout the 2 year duration of the study as they occur. ]

Central Contacts and Locations

Central contacts

Alfonso Iorio, MD, PhD, FRCPC

905-525-9140iorioa@mcmaster.ca

Noella Noronha, BSc

noronn@mcmaster.ca

Locations

University of Calgary

Recruiting

Calgary, Alberta, Canada

Contacts

Julia Hews-Girard, RN

julia.hewsgirard@albertahealthservices.ca

Principal Investigator:

Adrienne Lee, MD

University of Alberta

Recruiting

Edmonton, Alberta, Canada

Contacts

Vicki Voong, RN

vvoong@ualberta.ca

Principal Investigator:

Linda Sun, MD

University of Manitoba

Recruiting

Winnipeg, Manitoba, Canada

Contacts

Ming Yang

MYang@providencehealth.bc.ca

Principal Investigator:

Jayson Stoffman, MD

McMaster University

Recruiting

Hamilton, Ontario, Canada, L8S4B2

Contacts

Noella Noronha, BSc

noronn@mcmaster.ca

Principal Investigator:

Alfonso Iorio, MD

McMaster University

Recruiting

Hamilton, Ontario, Canada

Contacts

Theresa Almonte

almontet@hhsc.ca

Principal Investigator:

David Chan, MD

Queen's University

Recruiting

Kingston, Ontario, Canada

Contacts

Julie Grabell, MD

grabell@queensu.ca

Principal Investigator:

Paula James, MD

More Information

Sponsor

McMaster University

Last update posted

Mar 21, 2023

Last verified

Mar, 2023

Keywords

  • pharmacokinetics
  • popPK
  • PK
  • Hemophilia

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by McMaster University on 2023-03-21.