Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 158
- 试验地点
- 17
- 主要终点
- To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
研究概览
简要总结
Open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis.
Patients receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron.
After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.
详细描述
It has been widely shown that an appropriate chelating therapy in chronic anemias transfusion dependent can prevent the overstock of iron and can reduce the already existing overstock reducing, then, the co-morbidity and improving survival.
In particular, some authors have shown in MDS affected patients undergoing intensive chelating therapy with deferoxamine haematological recovery with a reduction of the need of transfusions.
With the present study, we plan to evaluate the safety and efficacy of a therapy with the new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis.
This is an open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis.
Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients, both males and females, with low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis.
- •Age >=18 years
- •Patients who never received chelation therapy or who received a therapy with Desferal after a day of wash out
- •Medical history of at least 20 blood transfusions (equivalent to 100 ml/kg of red cells concentrate).
- •Availability of data concerning blood transfusions during the 12 weeks before screening
- •Serum ferritin >= 1000 µg/L at least twice (at least 2 week interval between the 2 analysis) during the year before the screening
- •Life expectancy > 12 months
- •Availability of at least 3 complete blood counts (before transfusions) during the 12 weeks before the screening
排除标准
- •Diagnosis different from MDS (i.e. myelofibrosis)
- •Severe renal impairment (creatinine clearance < 60 ml/min)
- •ALT/AST > 500 U/L
- •Active B and/or C hepatitis
- •Patients treated during the past 4 weeks with experimental drugs for MDS (including thalidomide, azacitidine, arsenic trioxide). These patients become eligible after a "wash out" of at least 4 weeks
- •Concomitant treatment with another iron-chelating agent
研究组 & 干预措施
Deferasirox
干预措施: Deferasirox (Drug)
结局指标
主要结局
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
时间窗: On a monthly basis thereafter from baseline assessment.
次要结局
- To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels(At 3, 6, 9, and 12 months from baseline assessment.)
- Quality of Life evaluation.(At 3, 6, 9, and 12 months from baseline assessment.)
- Compliance to chelating therapy evaluation.(On a monthly basis thereafter from baseline assessment.)
- To evaluate the impact Deferasirox iron chelating therapy vs the normal demand of transfusions in a subgroup of pts that will not receive growth factors or chemotherapy according to their basal characteristics.(On a monthly basis thereafter from baseline assessment.)