Clinical trial NCT02796222: Factor Product Utilization and Health Outcomes in Patients With Hemophilia
Official title: Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A, Congenital
Hemophilia B, Congenital
Keywords: Hemophilia
Recombinant factor VIII
Recombinant factor VIII Fc
Recombinant factor IX
Patient-reported outcomes
Recombinant factor IX Fc
Lead sponsor: University of British Columbia
Sponsor class: OTHER
Collaborators: name: Biogen
First posted: 2016-06-10
Enrollment: 61
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility 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)

Primary outcomes:
measure: Change in the total annualized factor consumption (in units/kilogram/year)
timeFrame: From baseline to 24-month period on rFVIIIFc or rFIXFc
Secondary outcomes:
measure: Change in health-related quality of life (HRQoL) SF-36
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in health-related quality of life (HRQoL) Haem-A-Qol
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in health-related quality of life (HRQoL) CHO-KLAT
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in chronic pain Numeric Rating Scale (0-10)
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in chronic pain "Bodily Pain" subscale of SF-36
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in physical activity (IPAQ)
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in physical activity "Physical Functioning" subscale of SF-36.
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in mood "Mental Health" subscale of SF-36
timeFrame: From baseline to 3 months, 12 months and 24 months
measure: Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
timeFrame: From baseline to 3 months only
measure: Clinicians' and patients' reason for switching to rFVIIIFc
timeFrame: Baseline through study completion, an average of 2 years
measure: Clinicians' and patients' reason for switching to rFIXFc
timeFrame: Baseline through study completion, an average of 2 years
measure: Product used for treatment of breakthrough bleeding and surgical procedures
timeFrame: Baseline through study completion, an average of 2 years
measure: Total annualized number of factor infusions
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Annualized bleeding rate
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Ratio of annual factor consumption-to-annual factor prescription
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Number of infusions required to treat a breakthrough bleed
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Incremental factor utilization per joint bleed avoided
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
measure: Serious adverse events
timeFrame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: BC Hemophilia Adult Program
state: British Columbia
Study officials:
affiliation: Division of Hematology, Department of Medicine, University of British Columbia
name: Shannon Jackson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Division of Hematology/Oncology, Department of Pediatrics, University of Ottawa
name: Robert Klaassen, MD
role: STUDY_CHAIR
affiliation: Division of Hematology, Department of Medicine, University of Calgary
name: Man-Chiu Poon, MD
role: STUDY_CHAIR
affiliation: Division of Hematology, Department of Medicine, Dalhousie University
name: Sue Robinson, MD
role: STUDY_CHAIR
affiliation: BC Children's hospital, Division of Hematology, Department of Medicine, University of British Columbia
name: John Wu, MD
role: STUDY_CHAIR
affiliation: Hemophilia Program, Hamilton Health Services Program, McMaster University
name: Alfonso Iorio, MD
role: STUDY_CHAIR
affiliation: Hemophilia Program, St. Michael's Hospital, University of Toronto
name: Michelle Sholzberg, MD
role: STUDY_CHAIR
References: