Factor Product Utilization and Health Outcomes in Patients With Hemophilia

April 26, 2021 updated by: Shannon Jackson, University of British Columbia

Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes

Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
61

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6T 2G2
        • BC Hemophilia Adult Program

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Probability Sample

Study Population

This study will be conducted in male patients ≥12 years of age with severe and moderate hemophilia A or hemophilia B (baseline factor level <5%) who are able to sign the informed consent or assent.

Description

Inclusion Criteria:

  1. Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
  2. Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.

Exclusion Criteria:

  1. Unable or unwilling to provide informed consent
  2. Patients with an existing bleeding disorder other than hemophilia A or B
  3. History of hypersensitivity or severe allergic reactions to factor products
  4. Patients currently participating in a phase 1-3 study with another factor replacement product
  5. Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Hemophilia A patients on rFVIIIFc
Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc
Hemophilia A patients on rFVIII
Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII
Hemophilia B patients on rFIXFc
Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc
Hemophilia A patients on rFIX
Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change in the total annualized factor consumption (in units/kilogram/year)
Time Frame: From baseline to 24-month period on rFVIIIFc or rFIXFc
From baseline to 24-month period on rFVIIIFc or rFIXFc

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in health-related quality of life (HRQoL) SF-36
Time Frame: From baseline to 3 months, 12 months and 24 months
HRQoL will be measured using Short Form 36 (SF-36) in all patients
From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) Haem-A-Qol
Time Frame: From baseline to 3 months, 12 months and 24 months
Haem-A-QoL in patients over age 18 years
From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) CHO-KLAT
Time Frame: From baseline to 3 months, 12 months and 24 months
The Canadian Hemophilia Outcomes- Kids Life Assessment Tool (CHO-KLAT) in patients between ages 13-18 years
From baseline to 3 months, 12 months and 24 months
Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score
Time Frame: From baseline to 3 months, 12 months and 24 months
From baseline to 3 months, 12 months and 24 months
Change in chronic pain Numeric Rating Scale (0-10)
Time Frame: From baseline to 3 months, 12 months and 24 months
From baseline to 3 months, 12 months and 24 months
Change in chronic pain "Bodily Pain" subscale of SF-36
Time Frame: From baseline to 3 months, 12 months and 24 months
From baseline to 3 months, 12 months and 24 months
Change in physical activity (IPAQ)
Time Frame: From baseline to 3 months, 12 months and 24 months
Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ)
From baseline to 3 months, 12 months and 24 months
Change in physical activity "Physical Functioning" subscale of SF-36.
Time Frame: From baseline to 3 months, 12 months and 24 months
From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
Time Frame: From baseline to 3 months, 12 months and 24 months
From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Time Frame: From baseline to 3 months, 12 months and 24 months
Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
From baseline to 3 months, 12 months and 24 months
Change in mood "Mental Health" subscale of SF-36
Time Frame: From baseline to 3 months, 12 months and 24 months
partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
From baseline to 3 months, 12 months and 24 months
Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
Time Frame: From baseline to 3 months only
Partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
From baseline to 3 months only
Clinicians' and patients' reason for switching to rFVIIIFc
Time Frame: Baseline through study completion, an average of 2 years
Choice among list of common reasons for changing product
Baseline through study completion, an average of 2 years
Clinicians' and patients' reason for switching to rFIXFc
Time Frame: Baseline through study completion, an average of 2 years
Choice among list of common reasons for changing product
Baseline through study completion, an average of 2 years
Product used for treatment of breakthrough bleeding and surgical procedures
Time Frame: Baseline through study completion, an average of 2 years
Choice among list of products
Baseline through study completion, an average of 2 years
Total annualized number of factor infusions
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Annualized bleeding rate
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Ratio of annual factor consumption-to-annual factor prescription
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Number of infusions required to treat a breakthrough bleed
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Incremental factor utilization per joint bleed avoided
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Difference in annualized factor utilization between Fc and regular non-Fc prophylaxis, divided by the difference in annualized joint bleeding rate between the two groups.
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Serious adverse events
Time Frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of British Columbia

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Biogen

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Shannon Jackson, MD, Division of Hematology, Department of Medicine, University of British Columbia
  • Study Chair: Robert Klaassen, MD, Division of Hematology/Oncology, Department of Pediatrics, University of Ottawa
  • Study Chair: Man-Chiu Poon, MD, Division of Hematology, Department of Medicine, University of Calgary
  • Study Chair: Sue Robinson, MD, Division of Hematology, Department of Medicine, Dalhousie University
  • Study Chair: John Wu, MD, BC Children's hospital, Division of Hematology, Department of Medicine, University of British Columbia
  • Study Chair: Alfonso Iorio, MD, Hemophilia Program, Hamilton Health Services Program, McMaster University
  • Study Chair: Michelle Sholzberg, MD, Hemophilia Program, St. Michael's Hospital, University of Toronto

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 26, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 26, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 26, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 10, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 28, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 26, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CAN-FAB-15-10911

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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