Pharmacovigilance Evaluation Of Benefix (Registered) In Germany And Austria
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 80
- 试验地点
- 21
- 主要终点
- Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
详细描述
Non-interventional study: subjects to be selected according to the usual clinical practice of their physician
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
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with hemophilia B already receiving or starting treatment with reformulated BeneFIX®.
排除标准
- •Patients with hemophilia B treated with a product other than BeneFIX®.
- •Inclusion in the ongoing prospective registry of European hemophilia B patients using BeneFIX®.
研究组 & 干预措施
A
Patients with Hemophilia B
干预措施: BeneFIX (Drug)
结局指标
主要结局
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline until last visit (up to 8.7 years)
Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; cancer; congenital anomaly. AEs included both serious and non-serious. Relatedness to BeneFIX was assessed by the investigator.
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline until last visit (up to 8.7 years)
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 8.7 years) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious.
Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
时间窗: Baseline until last visit (up to 8.7 years)
FIX inhibitor development was defined as measured inhibitor titer of greater than (\>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay.
Number of Participants With Adverse Events (AEs) of Special Interest
时间窗: Baseline until last visit (up to 8.7 years)
An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Adverse Events of special interest included allergic reactions, less than expected therapeutic effect (LETE) of drug, lack of efficacy/low recovery, erythrocyte agglutination in tube or syringe red blood cell (RBC) agglutination phenomena and thrombogenicity.
Investigator Assessment of Treatment Tolerability of Participants
时间窗: End of study visit (any time up to 8.7 years)
Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor.
Participant Assessment of Treatment Tolerability
时间窗: End of study visit (any time up to 8.7 years)
Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor.
次要结局
- Mean Total Number of Bleeding Episodes in Participants(Baseline until last visit (up to 8.7 years))
- Mean Total Number of Bleeding Episodes Per Year in Participants(Baseline until last visit (up to 8.7 years))
- Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work(Baseline, up to 8.7 years)
- Investigator Assessment of Treatment Efficacy of Participants(End of study visit (any time up to 8.7 years))
- Investigator Assessment of Treatment Handling of Participants(End of study visit (any time up to 8.7 years))
- Assessment of Treatment Efficacy by the Participants(End of study visit (any time up to 8.7 years))
- Assessment of Treatment Handling by the Participants(End of study visit (any time up to 8.7 years))
- Investigator Assessment of Treatment Satisfaction of Participants(Baseline up to 8.7 years)