An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Biocad
- 入组人数
- 24
- 试验地点
- 19
- 主要终点
- Annualized bleeding rate (ABR) before and after a single dose of ANB-002
研究概览
简要总结
The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.
详细描述
After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject.
After completiong the lead-in period subjects will enter the main (interventional) period.
At the first visit of the main period subjects will recieve investigational product ANB-002.
The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Men diagnosed with hemophilia B aged 18 or older
- •FIX activity ≤2%
- •Absense of FIX inhibitor
- •≥150 previous exposure days of treatment with FIX concentrates
排除标准
- •Any diseases of blood and hematopoietic organs other than hemophilia B
- •A history of any gene therapy, including ANB-002
- •Diagnosed HIV-infection, not controlled with anti-viral therapy
- •Active HBV or HCV infection
- •Anti-AAV5 antibodies
- •Any active systemic infections or recurrent infections requiring systemic therapy
- •Any other disorders associated with severe immunodeficiency
- •Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder
- •Malignancies with less than 5 years of remission
研究组 & 干预措施
Lead-in
All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption
ANB-002 (arvenacogene sanparvovec)
After completion of lead-in period subjects will enter the main period which starts with ANB-002 (arvenacogene sanparvovec) infusion.
干预措施: ANB-002 (Genetic)
结局指标
主要结局
Annualized bleeding rate (ABR) before and after a single dose of ANB-002
时间窗: Lead-in period: at least 6 months prior to ANB-002 infusion (Lead-in arm); Month 12 to Month 18 post infusion (ANB-002 arm)
A comparison will be made between the mean ABR values in the lead-in period and within 12 months after stabilization of endogenous FIX activity, but not later than 18 months after the ANB-002 dose
次要结局
- FIX activity(18 months after drug administration; final assessment - 5 years)
- Annualized FIX consumption(18 months after drug administration; final assessment - 5 years)
- Proportion of subjects not receiving FIX prevention(18 months after drug administration)
- Annualized bleeding rate (ABR)(18 months after drug administration; final assessment - 5 years)
- Proportion of subjects with spontaneous bleedings(18 months after drug administration; final assessment - 5 years)
- Annualized rate of spontaneous bleedings(18 months after drug administration; final assessment - 5 years)
- Proportion of subjects with hemarthrosis(18 months after drug administration; final assessment - 5 years)
- Annualized hemarthrosis rate(18 months after drug administration; final assessment - 5 years)
- Proportion of subjects with bleedings requiring FIX concentrate therapy(18 months after drug administration; final assessment - 5 years)
- Annualized rate of bleedings requiring therapy with FIX concentrates(18 months after drug administration; final assessment - 5 years)
- Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity(12 months after stabilization of endogenous FIX activity)
- Proportion of subjects achieving clinical response(18 months after drug administration)
- Proportion of subjects achieving a normalized response(18 months after drug administration)