NCT05962398Enrolling By Invitation不适用
An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
适应症
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- CSL Behring
- 入组人数
- 56
- 试验地点
- 28
- 主要终点
- Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
研究概览
简要总结
The primary purpose of this study is to assess the long-term safety in male adults with hemophilia B who were treated with CSL222 in studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Treatment with CSL222 in Study CSL222_2001 or Study CSL222_
- •Provided written informed consent.
- •Willing and able to adhere to all protocol requirements.
排除标准
- 未提供
结局指标
主要结局
Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
时间窗: From Year 5 after administration of CSL222 in parent study up to Year 15
Number of SAEs and AESIs
时间窗: From Year 5 after administration of CSL222 in parent study up to Year 15
Percentage of Participants With SAEs and AESIs
时间窗: From Year 5 after administration of CSL222 in parent study up to Year 15
次要结局
- Annualized Bleeding Rate (ABR)(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Endogenous Factor IX (FIX) Activity(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Change from Baseline by Visit in Endogenous FIX Activity(At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15)
- Annualized Consumption of FIX Replacement Therapy(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Annualized Infusion Rate of FIX Replacement Therapy(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Number of Participants Remaining Free of Continuous FIX Prophylaxis(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Percentage of Participants Remaining Free of Continuous FIX Prophylaxis(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Number of New Target Joints(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Percentage Resolution of Pre-existing Target Joints(From Year 5 after administration of CSL222 in parent study up to Year 15)
- EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Change From Baseline in EQ-5D-5L Index Value(Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15)
- EQ-5D VAS Score(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Change From Baseline in EQ-5D VAS Score(Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15)
- Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score(From Year 5 after administration of CSL222 in parent study up to Year 15)
- Change From Baseline in Hem-A-QoL Total Score(Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15)
研究者
研究点 (28)
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