Factor Product Utilization and Health Outcomes in Patients With Hemophilia

Hemophilia A, Congenital Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02796222
• Other study ID #: CAN-FAB-15-10911
• Status: Completed
• Start date: April 2016
• Est. completion date: April 26, 2021

Study information

• Verified date: April 2021
• Source: University of British Columbia
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: April 26, 2021
• Est. primary completion date: April 26, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A, Congenital
• Hemophilia B
• Hemophilia B, Congenital

Locations

Country	Name	City	State
Canada	BC Hemophilia Adult Program	Vancouver	British Columbia

Sponsors (2)

Lead Sponsor	Collaborator
University of British Columbia	Biogen

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in the total annualized factor consumption (in units/kilogram/year)		From baseline to 24-month period on rFVIIIFc or rFIXFc
Secondary	Change in health-related quality of life (HRQoL) SF-36	HRQoL will be measured using Short Form 36 (SF-36) in all patients	From baseline to 3 months, 12 months and 24 months
Secondary	Change in health-related quality of life (HRQoL) Haem-A-Qol	Haem-A-QoL in patients over age 18 years	From baseline to 3 months, 12 months and 24 months
Secondary	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	From baseline to 3 months, 12 months and 24 months
Secondary	Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score		From baseline to 3 months, 12 months and 24 months
Secondary	Change in chronic pain Numeric Rating Scale (0-10)		From baseline to 3 months, 12 months and 24 months
Secondary	Change in chronic pain "Bodily Pain" subscale of SF-36		From baseline to 3 months, 12 months and 24 months
Secondary	Change in physical activity (IPAQ)	Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ)	From baseline to 3 months, 12 months and 24 months
Secondary	Change in physical activity "Physical Functioning" subscale of SF-36.		From baseline to 3 months, 12 months and 24 months
Secondary	Change in treatment satisfaction "Treatment" domain of Haem-A-QoL		From baseline to 3 months, 12 months and 24 months
Secondary	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.	From baseline to 3 months, 12 months and 24 months
Secondary	Change in mood "Mental Health" subscale of SF-36	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
Secondary	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.	From baseline to 3 months only
Secondary	Clinicians' and patients' reason for switching to rFVIIIFc	Choice among list of common reasons for changing product	Baseline through study completion, an average of 2 years
Secondary	Clinicians' and patients' reason for switching to rFIXFc	Choice among list of common reasons for changing product	Baseline through study completion, an average of 2 years
Secondary	Product used for treatment of breakthrough bleeding and surgical procedures	Choice among list of products	Baseline through study completion, an average of 2 years
Secondary	Total annualized number of factor infusions		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	Annualized bleeding rate		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	Ratio of annual factor consumption-to-annual factor prescription		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	Number of infusions required to treat a breakthrough bleed		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	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.	From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Secondary	Serious adverse events		From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)