A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
研究概览
简要总结
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
ICL670
Oral dose of ICL670 at 10 mg/kg daily
干预措施: ICL670 (Drug)
结局指标
主要结局
Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
时间窗: 12 months
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period
次要结局
- Change in Serum Ferritin Level.(Baseline, 12 Months)
- The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L(Week 28 and Week 52)
- Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)(Baseline, 12 month)
- Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)(Baseline, 12 month)