A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 21
- 主要终点
- Change in Hemoglobin from Baseline to Week 5
研究概览
简要总结
This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to <18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).
详细描述
Subjects will be randomized to treatment in a 2:1 ratio (ferumoxytol: iron sucrose) and stratified by age group (2 to <6 years; 6 to <12 years; and 12 to <18 years). Subjects will receive one of the following treatment regimens:
• Ferumoxytol: 7 mg Fe/kg IV (maximum 510 mg/dose) x 2 doses, the first dose administered on Day 1 and the second 2 to 8 days later.
OR
• Iron sucrose (Venofer®): 4 mg Fe/kg IV (maximum 200 mg/dose) x 5 doses, the first dose on Day 1 and subsequent doses administered at least once per week and up to 3 times/week. All subjects will be monitored at the study site through at least 1 hour after the completion of each infusion of study drug. Assessment of blood Hgb concentrations, adverse events, and other safety assessments will be performed through study Week 5.
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
- None
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female 2 years to <18 years of age at time of consent
- •Has IDA defined as:
- •Hemoglobin (Hgb) <11.0 g/dL AND
- •Any one or more of the following:
- •Transferrin saturation (TSAT) <20%
- •ferritin <100 ng/mL
- •Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate
排除标准
- •Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose
- •History of allergy to intravenous (IV) iron
- •History of ≥2 clinically significant drug allergies
- •Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)
- •Low systolic blood pressure (BP) (age 1 to 9 years <70 + [age in years x 2] mmHg, age 10 to 17 years <90 mmHg)
- •Hgb ≤7.0 g/dL
- •Serum ferritin level >600 ng/mL
研究组 & 干预措施
Ferumoxytol
Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron at a concentration of 30 mg Fe/mL, coated with polyglucose sorbitol carboxymethylether and formulated with mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution.
干预措施: ferumoxytol (Drug)
Iron sucrose
Each mL contains 20 mg of elemental iron as iron sucrose in water for injection. The 5 mL single-use vial contains 100 mg of iron per 5 mL. The drug product contains approximately 30% sucrose (300 mg/mL)
干预措施: Iron sucrose (Drug)
结局指标
主要结局
Change in Hemoglobin from Baseline to Week 5
时间窗: 35 days
Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5
次要结局
- Incidence of adverse events of special interest (AESI)(49 days)
- Incidence of Treatment Emergent Adverse Events(49 days)