An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 281
- 试验地点
- 79
- 主要终点
- Number of participants with treatment emergent adverse events (TEAEs)
研究概览
简要总结
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
-
To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:
-
Bleeding episodes
-
Spontaneous bleeding episodes
-
Joint bleeding episodes
-
To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
详细描述
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Fitusiran
Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
干预措施: Fitusiran (Drug)
结局指标
主要结局
Number of participants with treatment emergent adverse events (TEAEs)
时间窗: from study baseline (day 1) up to maximum 88 months
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
次要结局
- Annualized bleeding rate (ABR)(from first ever dose of fitusiran (day 1) up to maximum 78 months)
- Annualized joint bleeding rate(from first ever dose of fitusiran (day 1) up to maximum 78 months)
- Changes in Haem-A-quality of life (QoL) score(from first ever dose of fitusiran (day 1) up to maximum 78 months)
- Annualized spontaneous bleeding rate(from first ever dose of fitusiran (day 1) up to maximum 78 months)