An Open-label, One-center Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease
试验速览
- 阶段
- 3 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Proportion of patients with mean Hb ≥ 11.0 g/dL
研究概览
简要总结
This open-labeled, one-center study is designed to evaluate the efficacy and safety of roxadustat in ESA-naïve and ESA-treated pediatric patients with CKD Stages 3, 4, and 5 who are receiving dialysis or not. The study will enroll patients between the ages of 2 to <18 years. Approximately 30 patients will be enrolled.
详细描述
This study is to evaluate the use of roxadustat for renal anemia in pediatric patients with CKD
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
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Estimated glomerular filtration rate (Bedside Schwartz formula) of < 60 ml/min/1.73 m2 (stage 3, 4 and 5 CKD) for dialysis or non-dialysis patients.
- •For ESA-naïve patients, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be < 11.0 g/dL. For patients currently receiving stable ESA dosing who will discontinue ESA and convert to roxadustat during study, mean of two most recent central laboratory Hb values during the screening period obtained at least 2 days apart must be ≥ 9.0 g/dL and ≤ 12.5 g/dL
- •Ferritin >50 ng/mL and transferrin saturation >10%
排除标准
- •Uncontrolled hypertension as judged by the principal investigator prior to screening.
- •Known hematologic disease related anemia (including PRCA)
- •Known malignancy within the past 5 years before screening.
- •History of severe anaphylaxis or known allergic to ingredient of roxadusta.
- •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3x upper limit of normal (ULN) and total bilirubin (Tbili) ≤2x ULN(obtained from screening visit).
- •Serum folate and vitamin B12 > LLN and cannot be corrected (obtained from screening visit).
- •Any RBC transfusion during the past 4 weeks before screening.
- •Weight<10kg
- •Any prior exposure to roxadustat or any other HIF-PH inhibitor.
研究组 & 干预措施
Roxadustat
Starting doses of 20, 50, 70 or 100 mg/30,70,90,or 120mg based on weight and dialysis or not.
干预措施: Roxadustat (Drug)
结局指标
主要结局
Proportion of patients with mean Hb ≥ 11.0 g/dL
时间窗: weeks 16-24
After the use of roxadustat, the proportion of patients with mean Hb ≥ 11.0 g/dL
次要结局
- Mean change in Hb(weeks 16-24)
研究者
Mao Jianhua
Chief Physician
The Children's Hospital of Zhejiang University School of Medicine