A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 69
- 试验地点
- 7
- 主要终点
- Safety and tolerability
研究概览
简要总结
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.
详细描述
The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.
The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 25 additional evaluable subjects.
研究设计
- 研究类型
- Interventional
- 干预模型
- Sequential
- 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
- •Males and females aged ≥18 years;
- •ECOG performance status (PS) 0-2;
- •Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not eligible);
- •Received at least one prior systemic therapy, which may include stem cell transplant, for AL amyloidosis;
- •Have adequate organ function;
- •Ability to understand and willingness to sign informed consent prior to initiation of any study procedures.
排除标准
- •Secondary or familial amyloidosis;
- •Life expectancy of < 3 months;
- •Symptomatic multiple myeloma;
- •Hypersensitivities to other monoclonal antibodies;
- •Known HIV infection;
- •Women who are lactating;
- •Any other condition or prior therapy, which in the opinion of the PI, would make the subject unsuitable for the study.
研究组 & 干预措施
NEOD001
NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.
干预措施: NEOD001 (Drug)
结局指标
主要结局
Safety and tolerability
时间窗: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
* Adverse event profile * Dose limiting toxicity and maximum tolerated dose
Maximum tolerated dose
时间窗: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
* Adverse event profile * Dose Limiting Toxicity and maximum tolerated dose
次要结局
- Pharmacokinetics(28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator)
- Immunogenicity(28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator)