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Factor Product Utilization and Health Outcomes in Patients With Hemophilia

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ClinicalTrials.gov Identifier: NCT02796222
Recruitment Status : Recruiting
First Posted : June 10, 2016
Last Update Posted : June 20, 2019
Sponsor:
Collaborator:
Biogen
Information provided by (Responsible Party):
Shannon Jackson, University of British Columbia

Brief Summary:
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.

Condition or disease
Hemophilia A, Congenital Hemophilia B, Congenital

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Study Type : Observational
Estimated Enrollment : 120 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes
Actual Study Start Date : April 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort
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



Primary Outcome Measures :
  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 Outcome Measures :
  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) ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Criteria

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02796222


Contacts
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Contact: Ming Yang 1-604-682-2344 ext 63818 myang@providencehealth.bc.ca

Locations
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Canada, British Columbia
BC Hemophilia Adult Program Recruiting
Vancouver, British Columbia, Canada, V6T 2G2
Contact: Ming Yang    604-682-2344 ext 63818    myang@providencehealth.bc.ca   
Principal Investigator: Shannon Jackson, MD         
Sponsors and Collaborators
University of British Columbia
Biogen
Investigators
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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

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Responsible Party: Shannon Jackson, Clinical Associate Professor, University of British Columbia
ClinicalTrials.gov Identifier: NCT02796222     History of Changes
Other Study ID Numbers: CAN-FAB-15-10911
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: June 20, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Shannon Jackson, University of British Columbia:
Hemophilia
Recombinant factor VIII
Recombinant factor VIII Fc
Recombinant factor IX
Patient-reported outcomes
Recombinant factor IX Fc

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII
Coagulants