The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment
试验速览
- 阶段
- 4 期
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- AsBR
研究概览
简要总结
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location.
The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients with age ≥ 20 years old
- •Endogenous FIX activity ≤1 IU/dL,
- •At least 50 exposure days (EDs) with FIX products,
- •No detectable inhibitor to FIX or inhibitor history,
- •Had a minimum of 2 nontrauma-induced bleeding episodes (any type or location) treated in the 6 months preceding study entry,
- •The patient or patient's parents or legal authorized representative, as applicable, are capable to understand the study objectives and procedure, and sign the written consent,
- •Accept that the supply of Idelvion might be stopped once the study is completed,
- •Able to complete a diary during 12 months or 50 EDs, whichever comes first.
排除标准
- •Currently participating in an interventional clinical trial,
- •Known hypersensitivity to any FIX product or hamster protein,
- •Known inhibitor to FIX or inhibitor history,
- •With other comorbidities which are not suitable for this study, at investigator's discretion,
- •Not able to compliant with the prophylactic treatment,
- •Lacking previous treatment and bleeding records.
研究组 & 干预措施
Single arm_Idelvion treated
干预措施: Albutrepenonacog Alfa 1 UNT [IDELVION] (Drug)
结局指标
主要结局
AsBR
时间窗: 12 months
To evaluate AsBR of Idelvion in routine patient care
次要结局
未报告次要终点