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Personalized Medicine for Canadians With Hemophilia

NCT03615053 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 600

Last updated 2023-03-21

No results posted yet for this study

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

Interventions

OTHER

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.

Sponsors & Collaborators

Principal Investigators

  • Alfonso Iorio, MD, PhD, FRCPC · McMaster University

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-24
Primary Completion
2022-09-30
Completion
2023-09-30

Countries

  • Canada

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03615053 on ClinicalTrials.gov