Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Group
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Change in Serum Ferritin Values
研究概览
简要总结
Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.
详细描述
The secondary endpoints that were originally planned for this study were not analyzed as the study ended prematurely.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Main diagnosis: aplastic anemia
- •Absence of severe and/or uncontrolled comorbidities
- •Confirmed iron overload (serum ferritin ≥ 1000 mkg/L)
- •Serum creatinine is not higher than the upper limit of normal for the given age
- •Absence of severe proteinuria. Protein/Creatinine ratio should be < 0.5 mg/mg
- •Liver enzymes are < 5 ULN
- •Completion of a scheduled cycle of immunosuppressive treatment program, with no severe infectious or generalized hemorrhagic complications
- •WHO (ECOG) performance status ≤ 2
排除标准
- •No signed informed consent form
- •Patient is under 18 years old
- •Severe concomitant condition
- •Severe infectious and generalized haemorrhagic complication following regular planned cycle of programmed immune suppressive treatment.
- •History of increased sensitivity to active substance and any other ingredient of the medicinal product.
- •Creatinine clearance (CC) < 60 ml/min and/or creatinine concentration in blood serum is 2 or more times higher than upper limit of age normal by results of 2 tests at Visits 1 and
- •Severe liver disorders (class C by Child-Pugh scale).
- •Patients with aplastic anaemia in which chelator treatment will be ineffective due to rapid progression of the disease.
- •Significant proteinuria basing on protein creatinine ratio > 1.0 mg/ml in urine sample from second urination at Visits 1 and 2 (or as an alternative in 2 of 3 urine samples at screening);
- •Rare hereditary disorders related to galactose intolerance, severe deficit of lactase or glucose-galactose malabsorption;
- •Pregnancy, lactation;
- •Level of liver enzymes higher than 5 upper limits of age normal at Visits 1 and 2.
研究组 & 干预措施
Serum ferritin level ≥ 1,000 μg/l
Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study
干预措施: ICL670 (Drug)
Serum ferritin level ≥ 1,000 μg/l
Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study
干预措施: Chelation (Drug)
Serum ferritin level < 1,000 μg/l
Transfusion-dependent adult patients with AA and serum ferritin < 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product
干预措施: No chelation (Drug)
结局指标
主要结局
Change in Serum Ferritin Values
时间窗: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52
Change from baseline was be summarized descriptively for all on-treatment study visits. Changes to the planned statistical analysis were related to significant withdrawal of patients from the Per-Protocol Analysis Set due to a large number of patients who discontinued the study (lack of assessments of iron exchange parameters at visits) and deviations from the Protocol affecting the assessment of efficacy parameters. Because of that, the additional efficacy analysis in the Per-Protocol Analysis Set was not performed.
Change in Transferrin Saturation With Iron (TSI) Values
时间窗: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52
Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits.
Change in Serum Total Iron-binding Capacity (TIBC)
时间窗: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52
Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits.
次要结局
未报告次要终点