FORWARD: Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| rFVIIIFc for hemophilia A Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
Biological: rFVIIIFc
As described in the treatment arm
Other Names:
|
| 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 |
Biological: rFVIIIFc
As described in the treatment arm
Other Names:
Drug: non-Fc FVIII replacement products
Standard plasma-derived (pd) or recombinant (r) FVIII concentrate and other traditional treatment options
Other Names:
|
| rFIXFc for hemophilia B Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label |
Biological: rFIXFc
As described in the treatment arm
Other Names:
|
| 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 |
Biological: rFIXFc
As described in the treatment arm
Other Names:
Drug: non-Fc FIX replacement products
Standard plasma-derived (pd) or recombinant (r) FIX concentrate and other traditional treatment options
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product [Month 2 to month 14]
Secondary Outcome Measures
- 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]
TSQM V2 is a 11-item instrument designed to measure participants' satisfaction with medication within the past 2 to 3 weeks, or since their last medication use. Treatment satisfaction is assessed through side effects, effectiveness, convenience, and global satisfaction scale domains.
- Change in Hemophilia Activities List (HAL) [Baseline to 14 months]
HAL is an instrument designed to measure and quantify a participant's perceived functional ability to perform activities in daily life. The HAL consists of 42 multiple choice questions within seven domains: lying/sitting/kneeling/standing, function of legs, function of arms, use of transportation, self-care, household tasks, and leisure activities.
- Change in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-Pro) [Baseline to 14 months]
VERITAS-Pro is a 24-item questionnaire designed to measure treatment adherence through six sub-scales: Time, Dose, Plan, Remember, Skip, and Communicate. Each question is quantified on a 5-point Likert scale ("Always" to "Never") that correlates with a numeric score from 1 to 5, where higher scores indicate lower treatment adherence.
- Change in Wong-Baker Faces Pain Rating Scale [Baseline to 14 months]
Wong-Baker Faces Scale is a 1-item questionnaire consisting of six facial caricatures, each depicting a different level of current perceived pain intensity, ranging from a score of 0 to 10. Higher scores indicate greater pain.
- Change in Work Productivity and Impairment Questionnaire plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS) [Baseline to 14 months]
WPAI and classroom impairment questionnaire version is a 9-item questionnaire designed to measure hemophilia-related impairment in work, school, and activity level within the past 7 days. Visual analogue scales and fill-in responses are used to compute scores for absenteeism, impairment at work/school, losses in work/school productivity, and activity impairment based on scoring algorithms and presented as impairment percentages. Higher percentages indicate greater disease-related impairment
- Change in Missed planned activity and productivity [Baseline to 14 months]
The purpose of these free standing questions are to capture any missed activities due to hemophilia that are not captured in other questionnaires included in the study, especially among children and adolescents.
- Change in Caregiver burden [Baseline to 14 months]
The purpose of this survey is to assess how the intensive nature of caring for a hemophilia patient affects caregiver burden, providing scores on the following scales: Emotional burden, financial burden, lifestyle, social burden, and positive emotions. This will be completed by caregivers of children and adolescents younger than 18 years of age.
- Number of inpatient and outpatient healthcare visits [Baseline to 14 months]
Number of hospitalizations and number of emergency room visits or number of physician outpatient clinic visits
Eligibility Criteria
Criteria
Key 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
Key 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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Research Site | Los Angeles | California | United States | 90007 |
| 2 | Research Site | East Lansing | Michigan | United States | 48823 |
Sponsors and Collaborators
- Bioverativ Therapeutics Inc.
- Swedish Orphan Biovitrum
Investigators
- Study Director: Medical Director, Bioverativ Therapeutics Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 997HA401