An Open-label, Single-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Darbepoetin Alfa in Paediatric Subjects From Birth to Less Than 1 Year of Age With Anemia Due to Chronic Kidney Disease
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- Amgen
- 主要终点
- Number of subjects with clinically significant changes in physical examinations, laboratory safety tests, and vital signs
研究概览
简要总结
The purpose of this study is to find out more about darbepoetin alfa in children less than 1 year of age with anemia (a decrease in red blood cells) due to kidney failure. This study will see if darbepoetin alfa is safe and well tolerated and whether it causes any side effects by taking blood samples and checking vital signs (heart rate, body temperature, and blood pressure tests) at specific times throughout the study. In addition, the study will evaluate the amount of darbepoetin alfa in the blood over time and look at special markers in the blood to evaluate how darbepoetin alfa works on anemia.
Darbepoetin alfa is approved by the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) for use in adults, but not for all ages of pediatric subjects. Therefore, studies need to be conducted in pediatric subjects (children) to determine the appropriate dose to use in younger children.
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
- — 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Girls and boys between birth and < 1 year of age at the time of enrollment
- •Body weight ≥ 3 kg at screening and enrollment
- •Diagnosis of chronic kidney disease stage 3 to 5 with an estimated Glomerular Filtration Rate < 60 mL/min/1.73m2 without dialysis using the updated Schwartz Equation at screening; OR on dialysis at screening
- •Hemoglobin ≤ 9.0 g/dL within 7 days prior to enrollment
- •Transferrin saturation ≥ 20% at screening
排除标准
- •Premature girls and boys (< 37 weeks of gestation, counting from the first day of the mother's last menstrual period)
- •Peritoneal dialysis subjects with an episode of peritonitis within 30 days prior to enrollment
- •History of cardiovascular events or thromboembolism
- •History of upper or lower gastrointestinal bleeding
- •History of seizures
- •Active liver disease or history of liver disease
- •Uncontrolled hypertension defined as stage 2 hypertension or greater. This is defined as a systolic or diastolic blood pressure value greater than the 99th percentile + 5 mmHg for a subject's age
- •Major surgery 12 weeks prior to enrollment
- •Red blood cell transfusions 12 weeks prior to enrollment
- •Use of any erythropoiesis-stimulating agent within 12 weeks prior to enrollment
- •Currently receiving antibiotic therapy for systemic infection within 4 weeks prior to enrollment
- •Current or prior use of immunosuppressants (excluding low-dose corticosteroids, defined as ≤ 0.5 mg/kg per day prednisone or equivalent for ≤ 5 days)
- •Subject is receiving a dose higher than 0.5 mg/kg per day of prednisone (or equivalent dose of another corticosteroid) for > 5 days within 4 weeks prior to enrollment
- •Receiving or has received any investigational drug (or is currently using an investigational device) within the 30 days or 5 half-lives (whichever is longer) prior to enrollment
- •Subject has known hypersensitivity to darbepoetin alfa, r-HuEPO, or to any of the excipients
研究组 & 干预措施
Darbepoetin alfa
干预措施: Darbepoetin alfa (Drug)
结局指标
主要结局
Number of subjects with clinically significant changes in physical examinations, laboratory safety tests, and vital signs
时间窗: Assessed over 29 days
Number of subjects with treatment-emergent adverse events
时间窗: Assessed over 29 days
次要结局
- Change in transferrin saturation(Assessed from baseline to day 29)
- Maximum observed concentration (Cmax) of darbepoetin alfa(Assessed predose and at 6, 24, 48, 72, and 168 hours postdose)
- Time at which maximum concentration is observed (Tmax) of darbepoetin alfa(Assessed predose and at 6, 24, 48, 72, and 168 hours postdose)
- Change in reticulocytes(Assessed from baseline to day 29)
- Area under the concentration curve (AUC) of darbepoetin alfa(Assessed predose and at 6, 24, 48, 72, and 168 hours postdose)
- Terminal half-life (t½) of darbepoetin alfa(Assessed predose and at 6, 24, 48, 72, and 168 hours postdose)
- Clearance (CL) of darbepoetin alfa(Assessed predose and at 6, 24, 48, 72, and 168 hours postdose)
- Change in hemoglobin concentration(Assessed from baseline to day 29)
- Change in iron(Assessed from baseline to day 29)
- Change in ferritin(Assessed from baseline to day 29)