NCT01250951已完成4 期
1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 111
- 试验地点
- 1
- 主要终点
- changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
研究概览
简要总结
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
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
- •Age ≥ 2 years
- •Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)
- •ECOG Performance Status ≤ 2
- •Transfusion overload confirmed with ferritin level >1000 µg/l.
- •No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
- •Serum creatine level > ULN
- •No proteinuria
- •Liver enzymes level < 5 ULN.
- •No pregnancy or lactation
- •Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.
排除标准
- •Age < 2 years
- •No iron overload (Ferritin level <1000 µg/l).
- •Primary iron overload (hereditary hemochromatosis)
- •Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)
- •Elevated serum creatinine > ULN or/and proteinuria
- •Liver enzymes level >5 ULN.
- •Pregnancy or lactation.
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Deferasirox
Experimental
干预措施: Deferasirox (Drug)
结局指标
主要结局
changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade
时间窗: Baseline assessment is followed by monthly assessments for up to 1 year
次要结局
- changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade(at baseline and 1 at year (at the end of study).)
- Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.(From the start of study up to 1 year)
- changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination(Baseline assessment is followed by monthly assessments for up to 1 year.)
研究者
研究点 (1)
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