A Phase 2, Single Arm, Multicenter Study to Evaluate the Pharmacodynamics and Safety of Re-Treatment With ALXN2220 in Patients With Transthyretin Amyloid Cardiomyopathy
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 35
- 试验地点
- 6
- 主要终点
- Change from Baseline to Week 48 in ECV measured by cMRI
研究概览
简要总结
This is a Phase 2, single-arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in previous participants of Study NI006-101.
详细描述
This is a Phase 2, single arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in the patient population of previous participants in Study NI006-101.
The study will also explore changes in cardiac structure and function, and clinical function of this dosing regimen, as well as changes in cardiac amyloid load, structure and function during the treatment-pause since conclusion of Study NI006-101. The study consists of a Screening Period and a 48 week Active Treatment 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have received at least one dose of ALXN2220 in Study NI006-101
排除标准
- •Suspected or known intolerance/allergy to proteins or any components of the study drug
- •Treatment or study discontinuation in Study NI006-101 due to a treatment-related adverse event that was serious, severe or life-threatening (on Common terminology criteria for adverse events (CTCAE) scale)
- •Any new or uncontrolled condition after completion of Study NI006-101 that could make the participant unsuitable for participation in this study
研究组 & 干预措施
ALXN2220
Participants will receive ALXN2220 via intravenous (IV) infusion during the Active Treatment Period
干预措施: ALXN2220 (Drug)
结局指标
主要结局
Change from Baseline to Week 48 in ECV measured by cMRI
时间窗: Baseline, Week 48
Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy
时间窗: Baseline, Week 48
次要结局
- Incidence of TEAEs, including serious TEAEs(From Baseline up to Week 48)
- ALXN2220 serum concentrations(From Baseline up to Week 48)
- Incidence of Anti-Drug Antibodies(From Baseline up to Week 48)