A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 1,181
- 试验地点
- 217
- 主要终点
- Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
研究概览
简要总结
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.
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 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
- •End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
- •NT-proBNP > 2000 pg/mL at Screening
- •Treatment with a loop diuretic for at least 30 days prior to Screening
- •History of heart failure NYHA Class II-IV at Screening
- •Life expectancy of ≥ 6 months as per the Investigator's judgment
- •Males and females of childbearing ability must use contraception
排除标准
- •Known leptomeningeal amyloidosis
- •Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
- •Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
- •Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
- •LVEF < 30% on echocardiography
- •Renal failure requiring dialysis or an eGFR < 20 mL/min/1.73 m2 at Screening
- •Polyneuropathy with PND score IV
研究组 & 干预措施
Placebo
Participants will receive placebo via IV infusion q4w for at least 30 months up to a maximum of 48 months.
干预措施: Placebo (Drug)
ALXN2220
Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 30 months up to a maximum of 48 months.
干预措施: ALXN2220 (Drug)
结局指标
主要结局
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
时间窗: Baseline up to the end study (up to Month 48)
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
时间窗: Baseline up to the end of study (up to Month 48)
次要结局
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 30(Baseline, Month 30)
- Change from Baseline in Six-minute Walk Test (6MWT) at Month 30(Baseline, Month 30)
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24(Baseline, Month 24)
- Time to CV-related Mortality(Baseline up to the end of study (up to Month 48))
- Change from Baseline in Six-minute Walk Test (6MWT) at Month 24(Baseline, Month 24)
- Total Occurrences of CV Clinical Events(Baseline up to the end of study (up to Month 48))
- Time to ACM(Baseline up to the end of study (up to Month 48))