NCT00171171已完成3 期
A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Novartis
- 入组人数
- 252
- 试验地点
- 2
- 主要终点
- Liver Iron Concentration (as measured by biopsy)
研究概览
简要总结
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
- •Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone
排除标准
- •Means levels of ALT > 300 U/L
- •Serum creatinine above upper limit of normal
- •Active hepatitis C or chronic hepatitis B receiving specific treatment
研究组 & 干预措施
Deferasirox
Experimental
干预措施: deferasirox (Drug)
结局指标
主要结局
Liver Iron Concentration (as measured by biopsy)
时间窗: at baseline and after 1 yeor of ICL670 treatment
次要结局
- Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)(after 1 year of ICL670 treatment)
- Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation(monthly assessments for 1 year)
- Adverse events(during 1 year ICL670 treatment)
- hematology and biochemistry, urinalysis(monthly assessments for 1 year ICL670 treatment)
- ECG and Echocardiography(6-monthly for 1 year ICL670 treatment)
研究者
研究点 (2)
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