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Completed

NCT Number: NCT02796222

Factor Product Utilization and Health Outcomes in Patients With Hemophilia

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.

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Key information

Conditions

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

Age range

12 year and older

Sex eligibility

Male

Study type

Observational

Primary location

BC Hemophilia Adult Program

Vancouver, British Columbia, V6T 2G2, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
  • 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

  • Unable or unwilling to provide informed consent
  • Patients with an existing bleeding disorder other than hemophilia A or B
  • History of hypersensitivity or severe allergic reactions to factor products
  • Patients currently participating in a phase 1-3 study with another factor replacement product
  • 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)

Treatment and study plan

Primary outcomes

  1. Change in the total annualized factor consumption (in units/kilogram/year)

    Time frame: From baseline to 24-month period on rFVIIIFc or rFIXFc

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score

    Time frame: From baseline to 3 months, 12 months and 24 months

  5. Change in chronic pain Numeric Rating Scale (0-10)

    Time frame: From baseline to 3 months, 12 months and 24 months

  6. Change in chronic pain "Bodily Pain" subscale of SF-36

    Time frame: From baseline to 3 months, 12 months and 24 months

  7. 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)

  8. Change in physical activity "Physical Functioning" subscale of SF-36.

    Time frame: From baseline to 3 months, 12 months and 24 months

  9. Change in treatment satisfaction "Treatment" domain of Haem-A-QoL

    Time frame: From baseline to 3 months, 12 months and 24 months

  10. 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.

  11. 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.

  12. 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.

  13. 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

  14. 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

  15. 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

  16. 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)

  17. Annualized bleeding rate

    Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)

  18. 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)

  19. 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)

  20. 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.

  21. 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)

  22. Serious adverse events

    Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)

Sponsors and collaborators

Lead sponsor

University of British Columbia

Other

Collaborators

  • Biogen

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Jun 10, 2016
Registry last updated
Apr 28, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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