An observational study in Hemophilia A, Congenital and Hemophilia B, Congenital, sponsored by University of British Columbia. Completed at 1 site in Canada. Open to male participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2021-04-28.
Sponsored by University of British Columbia · Observational
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.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 61 is below the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →University of British Columbia is the lead sponsor of 1,309 studies on the registry; 253 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 1 (17%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc
Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII
Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc
Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX
Change in the total annualized factor consumption (in units/kilogram/year)
Time frame: From baseline to 24-month period on rFVIIIFc or rFIXFc
Change in health-related quality of life (HRQoL) SF-36
HRQoL will be measured using Short Form 36 (SF-36) in all patients
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) Haem-A-Qol
Haem-A-QoL in patients over age 18 years
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) CHO-KLAT
The Canadian Hemophilia Outcomes- Kids Life Assessment Tool (CHO-KLAT) in patients between ages 13-18 years
Time frame: 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
Change in chronic pain Numeric Rating Scale (0-10)
Time frame: 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
Change in physical activity (IPAQ)
Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ)
Time frame: 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
Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
Time frame: 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.
Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood "Mental Health" subscale of SF-36
partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
Partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Time frame: From baseline to 3 months only
Clinicians' and patients' reason for switching to rFVIIIFc
Choice among list of common reasons for changing product
Time frame: Baseline through study completion, an average of 2 years
Clinicians' and patients' reason for switching to rFIXFc
Choice among list of common reasons for changing product
Time frame: Baseline through study completion, an average of 2 years
Product used for treatment of breakthrough bleeding and surgical procedures
Choice among list of products
Time frame: 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)
Annualized bleeding rate
Time frame: 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)
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)
Incremental factor utilization per joint bleed avoided
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.
Time frame: 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)
Serious adverse events
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Plan to share: No
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This study is completed, as verified in Apr 2021. You cannot join it, but the record below documents what was studied.
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University of British Columbia