A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 129
- 试验地点
- 40
- 主要终点
- Number of Participants With Cardiac Response and Non-Response
研究概览
简要总结
This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Age ≥18 years
- •Confirmed diagnosis of systemic AL amyloidosis
- •≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response
- •Cardiac involvement
- •NT-proBNP ≥650
排除标准
- •Non-AL amyloidosis
- •Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma
- •NT-proBNP >5000
- •Received Plasma cell directed chemotherapy within 6 months
- •Received autologous stem cell transplant (ASCT) within 12 months
研究组 & 干预措施
NEOD001
Study Drug given IV every 28 days at 24mg/kg
干预措施: NEOD001 (Drug)
Placebo
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Cardiac Response and Non-Response
时间窗: Baseline through 12 months of treatment
N-terminal pro-brain natriuretic peptide (NT-proBNP ) best response (Response or Non-Response \[Stable, Progression\]) from baseline through 12 months of treatment. Cardiac best response, as assessed by NT-proBNP alone, is defined as the most favorable category among response (ie, decrease in NT-proBNP from baseline of \>30% and \>300 ng/L), stable (ie, neither response nor progression), and progression (ie, increase in NT-proBNP from baseline of \>30% and \>300 ng/L) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Non-response is defined as either stable or progression.
次要结局
- 6MWT Distance(Baseline to 12 months of treatment)
- SF-36v2 PCS Score(Baseline to 12 months of treatment)
- NIS-LL Total Score(Baseline to 12 months of treatment)
- NT-proBNP Slope(Baseline through 12 months of treatment)
- Hepatic Best Response(Baseline through 12 months of treatment)
- Number of Participants With Renal Best Response and Non-Response(Baseline through 12 months of treatment)