Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
研究概览
简要总结
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.
详细描述
This was a phase two pilot clinical trial designed to evaluate the safety and efficacy of the combination of deferasirox and deferoxamine in transfusion dependent thalassemia with a range of systemic iron burden. The duration of combined therapy was 12 months. Changes in liver iron concentration, ferritin, myocardial iron, and serum creatinine was monitored.
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
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Transfusion Dependent Thalassemia
- •If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding
- •Older than 8 years
排除标准
- •Participating on another interventional clinical trial
研究组 & 干预措施
Deferasirox (Exjade) and Deferoxamine (DFO)
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
干预措施: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO) (Drug)
结局指标
主要结局
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
时间窗: 12 months
Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
时间窗: 12 months
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
次要结局
未报告次要终点
研究者
Elliott Vichinsky
Director, Hematology/Oncology
UCSF Benioff Children's Hospital Oakland