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
- 不适用
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 9
- 试验地点
- 12
- 主要终点
- Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration
研究概览
简要总结
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).
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
- •- Subjects with congenital hemophilia B who completed Study CTAMT-060-01
- •- 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 CT-AMT-060-01.
结局指标
主要结局
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration
时间窗: 6-10 years after dosing in CT-AMT-060-01 study
Neutralizing FIX antibodies (FIX inhibitors)
时间窗: 6-10 years after dosing in CT-AMT-060-01 study
ALT/AST levels
时间窗: 6-10 years after dosing in CT-AMT-060-01 study
Liver pathology score Assessed by ultrasound every 6 months
时间窗: 6-10 years after dosing in CT-AMT-060-01 study
Alfa fetoprotein levels
时间窗: 6-10 years after dosing in CT-AMT-060-01 study
次要结局
- Total Consumption of FIX Replacement Therapy on-demand and prophylactic(6-10 years after dosing in CT-AMT-060-01 study)
- Number of Procedures (including major and minor surgeries)(6-10 years after dosing in CT-AMT-060-01 study)
- Endogenous Percent FIX activity(6-10 years after dosing in CT-AMT-060-01 study)
- Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds(6-10 years after dosing in CT-AMT-060-01 study)
- Quality of Life questionnaire EQ-5D-5L score(6-10 years after dosing in CT-AMT-060-01 study)
- Hemophilia Joint Health Score(6-10 years after dosing in CT-AMT-060-01 study)
- Quality of Life questionnaire SF-36 score(6-10 years after dosing in CT-AMT-060-01 study)