A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 9
- 试验地点
- 6
- 主要终点
- The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of CSL220.
研究概览
简要总结
To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of CSL220, an AAV vector containing a codon-optimized human coagulation hFIX gene, to adult subjects with severe or moderately severe haemophilia B.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 主要目的
- Long-term Follow-up
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects with congenital haemophilia B who completed Study CSL220_1001
- •Able to provide informed consent following receipt of verbal and written information about the trial
排除标准
- •Enrolled subjects will have already been assessed based on the exclusion criteria for Study CSL220_1001
结局指标
主要结局
The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of CSL220.
The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of CSL220.
Primary safety endpoints include the following: - AEs possibly or probably related to previous CSL220 administration
Primary safety endpoints include the following: - AEs possibly or probably related to previous CSL220 administration
Neutralising FIX antibodies (FIX inhibitors)
Neutralising FIX antibodies (FIX inhibitors)
ALT/aspartate aminotransferase (AST) levels
ALT/aspartate aminotransferase (AST) levels
Liver pathology (assessed by ultrasound)
Liver pathology (assessed by ultrasound)
Alpha-fetoprotein (AFP)
Alpha-fetoprotein (AFP)
次要结局
- The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of CSL220 on FIX activity, overall FIX utilisation, bleeding events, any procedures, and QoL.
- Secondary efficacy endpoints include the following: - Endogenous FIX activity
- Utilisation of FIX-replacement therapy
- Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds
- Procedures (including major and minor surgeries)
- 36-Item Short Form Survey (SF-36) and EQ-5D-5L Quality of Life (QoL) scores
- Hemophilia Joint Health Score (HJHS)
研究者
Trial Registration Coordinator
Scientific
CSL Behring LLC