An Open-Label Phase 2a Clinical Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Subjects With Primary IgA Nephropathy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Percent Reduction in 24-hour Urine Protein Excretion
研究概览
简要总结
The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.
详细描述
This is a Phase 2, single arm open-label clinical study in up to 25 participants that will consist of a screening period, a 24-week treatment period, an optional treatment extension period of up to an additional 48 weeks, and a 12- week post-treatment follow-up evaluation period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
IONIS-FB-LRx
干预措施: IONIS-FB-LRx (Drug)
结局指标
主要结局
Percent Reduction in 24-hour Urine Protein Excretion
时间窗: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)
次要结局
- Absolute Reduction in 24-hour Urine Protein Excretion(Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured))
- Absolute Reduction in Proteinuria (UPCr Ratio)(Baseline to Week 29)
- Percent Change from Baseline in Plasma Factor B (FB)(Up to Week 29)
- Percent Change from Baseline in Plasma AH50(Up to Week 29)
- Absolute Reduction in Albuminuria (UACr Ratio)(Baseline to Week 29)