An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 主要终点
- the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
研究概览
简要总结
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 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 71 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric patients aged from 2 to less than 6 years old.
- •Patients with transfusion dependent β-thalassemia major.
- •Serum ferritin values ≥ 1000 ng/ml at screening.
- •Written informed consent must be obtained from the patient's legal guardian in accordance with local law and regulation prior to any screening procedures.
排除标准
- •Non-transfusion-dependent thalassemia.
- •Systemic diseases which would prevent study treatment (e.g. uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.)
- •Serum creatinine > age adjusted ULN.
- •Significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 0.5mg/mg in a non-first void urine sample at screening. If UPCR is found to be ≥ 0.5 mg/mg the test can be repeated after 1 month.
- •ALT/AST > 2.5xULN and total bilirubin > 1×ULN.
- •Left ventricular ejection fraction < 56% by echocardiography.
- •Patient has a known history of HIV seropositivity or history of active/treated hepatitis B or C (a test for screening is not required).
- •A history of clinically relevant ocular and/or auditory toxicity related to iron chelation therapy
- •Any surgical or medical conditions which will significantly alter the absorption, distribution, metabolism or excretion of the drug(e.g. ulcerative disease, uncontrolled nausea, vomiting, malabsorption syndrome, obstruction, or stomach and/or small bowel resection).
- •Other conditions which investigator deems potential harm to patients if participate the study.
研究组 & 干预措施
Deferasirox
The study will provide PK, safety, tolerability and efficacy data collected during 48 weeks of treatment with deferasirox in Chinese pediatric patients with transfusion dependent -thalassemia major, aged 2 to <6 years at enrollment. The target patient pool consists of 20 patients with evidence of iron overload measured by serum ferritin level at the start of study. Patients will start their deferasirox treatment with a dose of 20 mg/kg/day. Serum ferritin will be monitored every month and the dose of deferasirox will be adjusted if necessary every 3 months based on the trends in serum ferritin. Other possible dose adjustments will be based on the patient's safety assessments.
干预措施: Deferasirox (Drug)
结局指标
主要结局
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
时间窗: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
Tmax was directly determined from the raw plasma concentration-time data.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.
时间窗: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
Area under the plasma concentration-time curve from time zero to the end of the dosing interval.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
时间窗: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
The maximum plasma concentration of study medication.
次要结局
- The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.(Baseline, every 4 weeks until 48 weeks after taking the drug)
- The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.(Baseline, every 4 weeks until 48 weeks after taking the drug)