A Multi-Center, Open-label, Non Comparative, Phase II Trial on Efficacy and Safety of ICL670 Given for 1 Year With Dose Adjustments Based on Serum Ferritin in Patients With Chronic Anemia and Transfusional Hemosiderosis Including an Additional 1 Year Extension.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 102
- 试验地点
- 1
- 主要终点
- Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1
研究概览
简要总结
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease.
During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Deferasirox
Participants received initial dose of 20 milligrams per kilogram (mg/kg) Deferasirox tablets was administered orally once daily (OD) based on the Participants body weight. The dose of Deferasirox was adjusted to either 10 mg/kg or 30 mg/kg based on the volumes of blood transfusions being administered.
干预措施: Deferasirox (ICL670) (Drug)
结局指标
主要结局
Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1
时间窗: From the Baseline, Year 1 (End of core study)
Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as \< 7, ≥ 7 to \< 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC.
次要结局
- Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years(From the Baseline, Year 2 (End of extension study))
- Correlation of LIC and Serum Ferritin at Core and Extension Study(From the Baseline, Year 1 (End of core study), Year 2 (End of extension study))
- Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup(From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study))
- Absolute Serum Ferritin Levels Over 2 Years(From the Baseline, Year 1 (End of core study), Year 2 (End of extension study))
- Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2(From the Baseline to End of Year 2 (End of extension study))
- Absolute Change From Baseline in Serum Ferritin Levels to Year 2(From the Baseline up to Year 2 (End of extension study))
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption(From the Baseline up to Year 2 (End of extension study))
- Number of Participants With Clinically Significant Ophthalmological Abnormalities(At 2 years (End of extension study))