An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 506
- 试验地点
- 7
- 主要终点
- Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
研究概览
简要总结
A 1-year randomized Phase III core trial (NCT00061750) using deferoxamine as the comparator was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older. Patients who successfully completed this main trial may continue in this extension trial to receive chelation therapy with deferasirox for an additional 4 years.
The objective of this study is to assess the efficacy and long-term safety of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Deferasirox
All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
时间窗: up to 5 years
Adverse events results are based on preferred terms with at least 7% of participants in any group.
次要结局
- Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))
- Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study(Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years))