Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
UNKNOWN
NA
600 participants
INTERVENTIONAL
2019-07-24
2023-09-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Web-based Application for the Population Pharmacokinetic Service - Phase 1
NCT02061072
Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability
NCT03533504
Evaluation of a Simple Pharmacokinetic Tool (myPKFiT™) to Guide Personalized Factor VIII Dosing in Patients With Hemophilia
NCT02750085
ATHN 7: Hemophilia Natural History Study
NCT03619863
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
NCT02585635
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
The study start date at each centre are staggered by 1-3 months in a modified wedge-shaped design, to allow better differentiation of the effect of the intervention from unrelated but concomitant changes in other aspects of care in the Canadian landscape. Outcomes of interest will be measured for one year prior and for one year after the implementation of the WAPPS-Hemo regimen tailoring procedure.
The two main objectives of this study are:
1. Evaluate the applicability and effectiveness of WAPPS-Hemo PopPK-based tailoring of factor concentrate regimens.
2. Generate a solid base of data to model the bleeding risk of severe hemophilia A/B patients on prophylaxis, evaluating the contribution of patient and treatment characteristics to individual bleeding risk to be reduced by the tailored prophylaxis approach.
PMCH will objectively measure the impact of adopting a PopPK based tailoring of hemophilia treatment. The first goal will be minimizing the occurrence of bleeding events in the hemophilia population. The bleeding rate of Canadian hemophilia patients is still measurable at 2-4 spontaneous joint bleeds per year, which in turn reduce quality of life and consume health resources. It is expected that optimizing treatment goal and modalities will reduce this burden, or at least will not increase it, allowing the pursuit the second goal: minimize the use of resources and prompt a more equitable distribution of factor concentrates. For some patients, standard prophylaxis dosing leads to excessive use of concentrates. It is expected that a small but sizeable proportion of the patient population will be able to successfully reduce their factor concentrate consumption. The third goal will be to generate an evidence-based approach to identify the appropriate target goal(s) for individual patients by modelling the components of their risk of bleeding. Adopting a variable target threshold may enhance objective one and two, maximizing benefits with appropriate allocation of resources.
In addition to WAPPS-Hemo, the study will involve two other tools available to hemophilia treaters in Canada: the Canadian Bleeding Disorders Registry (CBDR) and Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. In particular, this study will leverage the integration of the three tools described above: WAPPS-Hemo, with its ability to generate PK estimates and use them to optimize treatment regiments; CBDR, already collecting most of the information needed to both generate and estimate optimal regimen recommendations for tailoring an individual regimen to meet specified treatment goals; and PROBE, to measure the impact on patient critical life experiences and outcome.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
OTHER
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Tailored Regimen
Implementation of WAPPS-Hemo personalized dosing regimen.
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.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* on continuous factor prophylaxis;
* must be registered on CBDR (iCHIP in BC)
Exclusion Criteria
* another congenital or acquired bleeding disorders other than Hemophilia A or B;
* active inhibitors (\> 5 Bethesda units) or currently undergoing immune tolerance induction.
6 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
McMaster University
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Alfonso Iorio, MD, PhD, FRCPC
Role: PRINCIPAL_INVESTIGATOR
McMaster University
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Calgary
Calgary, Alberta, Canada
University of Alberta
Edmonton, Alberta, Canada
University of British Columbia
Vancouver, British Columbia, Canada
University of Manitoba
Winnipeg, Manitoba, Canada
McMaster University
Hamilton, Ontario, Canada
McMaster University
Hamilton, Ontario, Canada
Queen's University
Kingston, Ontario, Canada
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
PMCH Study
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.