An Open-label Phase 1 Safety Study of SANGUINATE™ Infusion in Patients With Acute Severe Anemia Who Are Unable to Receive Red Blood Cell Transfusion
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 103
- 试验地点
- 32
- 主要终点
- Number and severity of adverse events within 24-hours of each SANGUINATE infusion
研究概览
简要总结
In this open-label study, eligible and consenting adult patients admitted to the hospital for critical care that have acute severe anemia, defined as a blood hemoglobin level ≤ 5 g/dL, but who are unwilling or unable to receive red blood cell (RBC) transfusion, will receive one or more infusions of SANGUINATE as are deemed necessary by the Investigator for survival of the acute anemic episode.
详细描述
Data collection for each patient during the in-patient portion of the study will be required for up to a maximum of 14 days while under critical care, with additional collection of safety-related findings at 7 days and 14 days after the last infusion of SANGUINATE. Each patient will thus have up to 28 days participation in the trial.
A minimum of 100 subjects are to be enrolled and to receive at least one infusion of SANGUINATE, with 14-day follow-up after the last infusion.
Because of the life-threatening effects of severe anemia, it is expected that all patients in this trial will receive care within a critical care facility (may be called intensive care or some similar name), wherein all patients will have real-time monitoring of vital signs and ECG (e.g., by telemetry) before, during, and after all study drug infusions.
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
- •The patient would otherwise receive RBC transfusion for treatment of severe anemia but cannot (hemolytic/allosensitized or no compatible blood) or will not (religious/personal objection) receive RBCs
- •Hemoglobin ≤ 5 g/dL, or hemoglobin ≤ 7 g/dL following a decline of ≥ 5 g/dL over less than 7 days
- •Age ≥ 18 years
- •Receiving or willing to receive supplemental iron therapy (unless contraindicated)
- •Receiving or willing to receive erythropoiesis-stimulating agent (EPO) therapy
- •Patient or legally authorized representative provided consent to participate
- •Investigator determination that the patient is an appropriate candidate for study enrollment
排除标准
- •Hemoglobin ≤ 2 g/dL
- •Presence of severe trauma (e.g., Injury Severity Score (ISS)/New Injury Severity Score (NISS) Score ≥ 25)
- •Unable to provide sufficient blood sample volume for screening assessments; or
- •Pregnant; or
- •Investigator determination that the patient is not an appropriate candidate for study enrollment
研究组 & 干预措施
SANGUINATE™
As Needed Dosing of SANGUINATE
干预措施: SANGUINATE™ (Drug)
结局指标
主要结局
Number and severity of adverse events within 24-hours of each SANGUINATE infusion
时间窗: 24 Hours
Composite endpoint defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis, death and other reported adverse events
次要结局
- Percentage of patients who recover from hypoxia-induced abnormal end-organ function(14 Days)
- Percentage of patients who improved with their hemodynamic status(14 Days)
- Percentage of patients who discontinue inotrope therapy(14 Days)
- Percentage of patients who discontinue respiratory support/therapy(14 Days)
- Number of serious adverse events following the start of SANGUINATE infusions through the final visit (up to 28 days)(28 Days)
- Percentage of patients who survive the acute episode of severe anemia(14 Days)