jRCT2033230266进行中（未招募）不适用
Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy and Safety of a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, CSL222) Administered to Japanese Adult Male Subjects with Severe or Moderately Severe Hemophilia B
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 4
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomized Controlled Trial
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 18age old over 至 No limit（—）
- 性别
- Male
入选标准
- •Assigned male at birth, age >= 18 years, of Japanese origin
- •Subject with congenital hemophilia B with known severe or moderately severe FIX deficiency (<= 2% of normal circulating FIX) for which the subject is on continuous routine FIX prophylaxis
- •> 150 previous exposure days of FIX replacement therapy
排除标准
- •History of FIX inhibitors
- •Positive human immunodeficiency virological test
- •Hepatitis B or C infection
- •Known coagulation disorder other than hemophilia B
结局指标
主要结局
-
Annualized bleeding rate (ABR) for CSL222 and Lead-in FIX prophylaxis.
次要结局
未报告次要终点
研究者
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