An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 Year) With Iron Deficiency Anemia
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 4
- 主要终点
- Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
研究概览
简要总结
An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.
详细描述
A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.
Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
- •Screening Hb ≥7 g/dL to <10 g/dL.
- •Infants with any of the following conditions:
- •Heart failure with IDA defined as syndromes of excessive preload, excessive afterload, abnormal rhythm, or decreased contractility
- •Gastrointestinal diseases with acquired short bowel syndrome (due to volvulus, necrotizing enterocolitis from surgical resection or spontaneous intestinal perforation)
- •Gastrointestinal intolerance of oral iron or an unsatisfactory response to oral iron
- •Other conditions associated with IDA which in the opinion of the investigator might benefit from administration of FCM
排除标准
- •Known history of hypersensitivity reaction to FCM.
- •Body weight <2.5 kg.
- •History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
- •Hemodialysis-dependent chronic kidney disease.
- •History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant at added risk for participation in the study.
- •Active infection.
- •Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
- •Blood transfusion in the 4 weeks prior to consent.
- •Administration of an iron-containing product within 14 days of administration of the study article.
- •Administration and / or use of an investigational product (drug or device) within 30 days of screening.
- •Current participation in another clinical trial.
- •Unable to comply with study procedures and assessments.
研究组 & 干预措施
Ferric Carboxymaltose
To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 5.0 mg/kg dose of FCM
干预措施: Ferric carboxymaltose (Drug)
Injectafer
To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 7.5 mg/kg dose dose of FCM.
干预措施: Ferric carboxymaltose (Drug)
结局指标
主要结局
Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
时间窗: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Change in reticulocytes count: %
时间窗: baseline to Days 8, 15, 22, and 36
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Change in hemoglobin (Hb): g/dL
时间窗: baseline to Days 8, 15, 22, and 36
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Treatment-emergent adverse events
时间窗: Baseline to Day 36
Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
Evaluate the PD parameters - Change in serum iron: mcg/dL
时间窗: baseline to Day 36
To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Evaluate the PD parameters - Change in serum ferritin: ng/mL
时间窗: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL
时间窗: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
次要结局
未报告次要终点