An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 500
- 试验地点
- 13
- 主要终点
- Bleeding Rate (all bleeds) - HEMGENIX Cohort
研究概览
简要总结
This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
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
- All
- 接受健康志愿者
- 否
入选标准
- •HEMGENIX Cohort:
- •- Treatment with commercial HEMGENIX.
- •- Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.
- •FIX Prophylaxis Cohort:
- •- Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.
排除标准
- •HEMGENIX Cohort:
- •- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
研究组 & 干预措施
HEMGENIX
Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
干预措施: HEMGENIX (Genetic)
FIX Prophylaxis
Patients with hemophilia B on FIX prophylaxis and enrolled in American Thrombosis and Hemostasis Network (ATHN) Transcends (A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People with Non-Neoplastic Hematologic Disorders) Hemophilia Cohort, or a similar registry.
干预措施: Factor IX (FIX) (Biological)
结局指标
主要结局
Bleeding Rate (all bleeds) - HEMGENIX Cohort
时间窗: During the 52 weeks following stable FIX expression (6 to 18 months) in the follow-up period and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
Annualised Bleeding Rate (ABR) for all bleeds
次要结局
- Zero bleeds - HEMGENIX Cohort(During follow-up after HEMGENIX treatment, up to 180 months)
- Related Serious Adverse Events (SAEs) - number(During 15 years of follow-up)
- Adverse Events of Special Interest (AESIs) - number(During 15 years of follow-up)
- Bleeding Rate (specified bleed types) - HEMGENIX Cohort(During follow-up period (52 weeks following stable FIX expression [6 to 18 months]), and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.)
- FIX activity - HEMGENIX Cohort(Day 1, Week 6, and Months 6, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.)
- Annualized consumption of FIX replacement therapy - HEMGENIX Cohort(During the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], & at Months 0 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180), & at yearly intervals (ie, at 0 to 1 yr, 1 to 2 yrs, etc, up to 14 to 15 yrs).)
- Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort(At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.)
- Target joints - FIX Prophylaxis Cohort(From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.)
- Related SAEs - percent(During 15 years of follow-up)
- Correlation analysis - HEMGENIX Cohort(Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.)
- Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort(At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.)
- AESIs - percent(During 15 years of follow-up)
- Bleeding Rate - FIX Prophylaxis Cohort(In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.)
- Target joints occurrence - HEMGENIX Cohort(After HEMGENIX treatment, during the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180).)
- Target joints resolution - HEMGENIX Cohort(During the follow-up period, up to 180 months)