A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes
Overview
- Phase
- N/A
- Intervention
- rFVIIIFc
- Conditions
- Hemophilia A
- Sponsor
- Bioverativ Therapeutics Inc.
- Enrollment
- 3
- Locations
- 1
- Primary Endpoint
- Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
- Status
- Terminated
- Last Updated
- 5 years ago
Overview
Brief Summary
The primary objective of the study is to evaluate the effectiveness of prophylactic treatment with recombinant Factor VIII Fc fusion protein (rFVIIIFc) and recombinant Factor IX Fc fusion protein (rFIXFc) therapy as assessed by patient treatment burden and health economic outcomes while maintaining disease control in males with hemophilia A or B.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Have a medically documented diagnosis of hemophilia A or B that is being treated prophylactically with a factor replacement product not designed to have a prolonged half-life and satisfy a therapeutic indication for rFVIIIFc/rFIXFc per the approved local label
- •Have at least 50 prior exposure days (EDs) to any combination of factor replacement products
- •Have documented pre-study data available that confirm fulfillment of the eligibility criteria
- •Have no measurable inhibitor activity in a sample obtained within 4 weeks prior to the Baseline visit, and absence of clinical signs or symptoms of decreased response to the current factor replacement product
Exclusion Criteria
- •Have a diagnosis of any bleeding disorder other than hemophilia A or hemophilia B or an additional coagulation disorder(s) in addition to hemophilia A or hemophilia B
- •Have a prior history of anaphylaxis associated with any factor VIII (FVIII)/ factor IX (FIX) or intravenous immunoglobin administration
- •Had an inhibitor within 5 years before the Baseline visit. Note: A family history of inhibitors will not exclude the patient.
- •Past or current treatment with any factor replacement product with a prolonged half-life, including an Fc product, for the treatment of hemophilia
- •NOTE: Other Protocol Defined Inclusion/ Exclusion Criteria May Apply.
Arms & Interventions
rFVIIIFc for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: rFVIIIFc
non-Fc (fusion protein) replacement products for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: rFVIIIFc
non-Fc (fusion protein) replacement products for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: non-Fc FVIII replacement products
rFIXFc for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: rFIXFc
non-Fc factor replacement products for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: rFIXFc
non-Fc factor replacement products for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
Intervention: non-Fc FIX replacement products
Outcomes
Primary Outcomes
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
Time Frame: Month 2 to month 14
Secondary Outcomes
- Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS)(Baseline to 14 months)
- Number of inpatient and outpatient healthcare visits(Baseline to 14 months)
- The total annualized factor consumption (in International Units [IU] per kilogram [IU/kg]) calculated for each participant(Month 2 to month 14)
- Change in Treatment Satisfaction Questionnaire for Medication (TSQM)(Baseline to 14 months)
- Change in Hemophilia Activities List (HAL)(Baseline to 14 months)
- Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro)(Baseline to 14 months)
- Change in Wong-Baker Faces Pain Rating Scale(Baseline to 14 months)
- Change in Caregiver burden(Baseline to 14 months)
- Change in Missed planned activity and productivity(Baseline to 14 months)