A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 260
- 试验地点
- 78
- 主要终点
- Time to Composite of All-cause Mortality or Cardiac Hospitalization
研究概览
简要总结
This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached.
详细描述
This is a multi-center, international, randomized, double-blind, placebo-controlled, two-arm efficacy and safety study in subjects newly diagnosed with AL amyloidosis. Approximately 236 subjects will be enrolled in ~60 centers, with approximately 118 subjects per arm.
This is an event driven trial, therefore subjects will remain on-study until study completion, which will occur when all primary endpoint events (all-cause mortality or cardiac hospitalizations) have been reached. All subjects who discontinue will be followed until the last event is adjudicated. The estimated overall study duration is approximately 42 months, including the enrollment and treatment periods
Study drug will be administered once every 28 days as a 60-120 minute IV infusion.
First-line chemotherapy must be a bortezomib-containing regimen, with bortezomib administered weekly. The number of cycles of first-line chemotherapy that are administered are at the discretion of the Investigator, and subsequent chemotherapy regimens may be prescribed as per standard of care at the Investigator's discretion.
An independent Data Monitoring Committee (DMC) will review data on a regular basis.
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
- •Newly diagnosed, AL amyloidosis treatment naïve
- •Bone marrow consistent with plasma cell dyscrasia
- •Confirmed diagnosis of AL amyloidosis
- •Cardiac involvement
- •Planned first-line chemotherapy contains a proteasome-inhibiting agent administered weekly
- •Adequate bone marrow reserve, hepatic and renal function
排除标准
- •Non-AL amyloidosis
- •Meets diagnostic criteria for symptomatic multiple myeloma
- •Subject is eligible for and plans to undergo ASCT
- •History of Grade ≥ 3 infusion-associated AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine or acetaminophen
研究组 & 干预措施
NEOD001
24 mg/kg (maximum dose of 2500 mg) of NEOD001 administered once every 28 days.
干预措施: NEOD001 (Drug)
Placebo
Placebo will be administered as a 250 mL bag of normal saline once every 28 days.
干预措施: Placebo (Other)
结局指标
主要结局
Time to Composite of All-cause Mortality or Cardiac Hospitalization
时间窗: Randomization until the date of death or cardiac hospitalization, up to 32 months
Time to all-cause mortality death occurring after the first infusion of study drug or cardiac hospitalization as adjudicated by the CEC occurring at least 91 days after first infusion of study drug through last subject last visit, whichever came first
次要结局
未报告次要终点