Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 184
- 试验地点
- 6
- 主要终点
- The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
研究概览
简要总结
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.
The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.
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
- •Patients completed the planned 12-month core study
- •Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
- •Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation
排除标准
- •Pregnant or breast feeding patients
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Deferasirox
Deferasirox daily oral dose between 5-40 mg/kg/day
干预措施: Deferasirox (Drug)
结局指标
主要结局
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
时间窗: Core study Baseline to the end of the study (up to 60 months)
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
次要结局
- The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study(Core study Baseline to end of extension study (up to 60 months))
- The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study(Core study Baseline to end of extension study (up to 60 months))
- The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study(Core study Baseline to end of extension study (up to 60 months))