A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
研究概览
简要总结
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
- •Patients currently participating in the food-effect sub-study, according to amendment
- •Ability to provide written informed consent prior to participation in this non-comparative extension study.
- •Female patients 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.
- •Body weight of at least 35 kg.
排除标准
- •Pregnant or breastfeeding patients.
- •History of non-compliance to medical regimens and patients who are considered potentially unreliable.
- •Proteinuria > 300 mg/L second void morning urine.
- •Patients with serum creatinine above the upper limit normal.
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Deferasirox
Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study
干预措施: Deferasirox (Drug)
Deferasirox Crossover
Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study
干预措施: Deferasirox (Drug)
结局指标
主要结局
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
时间窗: Baseline to 7 Years
The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.
次要结局
- Relative Change in Serum Ferritin From Baseline to 3.5 Years(Baseline to 3.5 years)