Personalized Medicine for Canadians With Hemophilia

NCT03615053 | Unknown

• 1 other identifier: PMCH Study
• Study Type: interventional
• Target: 600
• Locations: 1 country
• Sites: 7

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

Conditions

Hemophilia A; Hemophilia B

Keywords

pharmacokinetics; popPK; PK; Hemophilia

Outcome Measures

Primary Outcomes (2)

• Change in patient quality of life

  Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. Scores range from 0-1, with a higher value indicating better health status.

  Completed every 3-6 months for duration of the study, from enrollment to study completion.

• Annualized Bleeding Rate (ABR) pre and post-tailoring implementation

  ABR from the one year prior to WAPPS-Hemo tailoring to the one-year post-tailoring (absolute number of bleeds per year).

  Recorded throughout the 2 year duration of the study as they occur.

Secondary Outcomes (7)

• Change in physical activity

  Completed every 3-6 months for duration of the study, from enrollment to study completion.

• Adherence to prescribed regimen

  Recorded throughout the 2 year duration of the study - frequency is as input by patient.

• Consumption of factor concentrates

  Recorded throughout the 2 year duration of the study - frequency as input by patient.

• Feasibility and acceptability of the WAPPS-Hemo based prophylaxis tailoring

  Recorded after 1 year at the time of tailoring implementation.

• Characteristics of reported bleeds

  Recorded throughout the 2 year duration of the study- frequency as input by patient.

Study Arms (1)

Tailored Regimen

OTHER

Implementation of WAPPS-Hemo personalized dosing regimen.

Other: Implementation of WAPPS-Hemo personalized dosing regimen

Interventions

Implementation of WAPPS-Hemo personalized dosing regimen OTHER

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.

Tailored Regimen

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• McMaster University: lead

Study Sites (7)

University of Calgary

Calgary, Alberta, Canada

RECRUITING

University of Alberta

Edmonton, Alberta, Canada

RECRUITING

University of British Columbia

Vancouver, British Columbia, Canada

NOT YET RECRUITING

University of Manitoba

Winnipeg, Manitoba, Canada

RECRUITING

McMaster University

Hamilton, Ontario, L8S4B2, Canada

RECRUITING

McMaster University

Hamilton, Ontario, Canada

RECRUITING

Queen's University

Kingston, Ontario, Canada

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Alfonso Iorio, MD, PhD, FRCPC

  McMaster University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alfonso Iorio, MD, PhD, FRCPC

CONTACT

905-525-9140; iorioa@mcmaster.ca

Noella Noronha, BSc

CONTACT

noronn@mcmaster.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The PMCH study is a Canadian multicentre, open-label, historically-controlled clinical trial to evaluate the effects of implementing WAPPS-Hemo PopPK-based tailoring of hemophilia prophylaxis regimens using the tailoring dosing function of the WAPPS-Hemo system (WAPPS-Hemo clinical calculator). The study has a non-inferiority design for the primary efficacy and safety outcomes.

Study Record Dates

• First Submitted: July 18, 2018
• First Posted: August 3, 2018
• Study Start: July 24, 2019
• Primary Completion: September 30, 2022
• Study Completion: September 30, 2023
• Last Updated: March 21, 2023

Record last verified: 2023-03

Locations

CA (7)