A Global, Multicenter, Observational Study Evaluating the Impact of rFVIIIFc and rFIXFc on Patient-reported Treatment Burden and Health Economic Outcomes
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
rFVIIIFc for hemophilia A
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
干预措施: rFVIIIFc (Biological)
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
干预措施: rFVIIIFc (Biological)
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
干预措施: non-Fc FVIII replacement products (Drug)
rFIXFc for hemophilia B
Administered based on the clinical judgment of the Prescribing Physician and according to the local approved drug label
干预措施: rFIXFc (Biological)
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
干预措施: rFIXFc (Biological)
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
干预措施: non-Fc FIX replacement products (Drug)
结局指标
主要结局
Annualized number of injections for prophylactic treatment with a Factor VIII or Factor IX replacement product
时间窗: Month 2 to month 14
次要结局
- 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)