NCT01709838已完成4 期
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 134
- 试验地点
- 1
- 主要终点
- Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
研究概览
简要总结
Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment. Provided further assessment of the long-term efficacy and safety of deferasirox in NTDT patients with iron overload (LIC ≥ 5 mg Fe/g liver dw and SF ≥ 300 ng/mL) for up to 260 weeks.
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
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Non-transfusion dependent congenital or chronic anemia inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease)/ Liver iron concentration >/= 5 mg Fe/g dw Serum Ferritin >/= 300 ng/mL
排除标准
- •HbS-beta Thalassemia, anticipated regular transfusion program during the study, blood transfusion 6 months prior to study start, significant proteinuria, creatinine clearance </= 40 ml/min, serum creatinine > ULN, ALT >5 x ULN, active hepatitis B or C, cirrhosis
- •Pediatrics Only:
- •A patient's weight of at least 20 kg is required to allow dosing of 5 mg/kg with one tablet of 125 mg
研究组 & 干预措施
Deferasirox
Other
All patients were treated with 10mg/kg/day deferasirox with dose adjustments after 4 weeks of treatment according to baseline Liver Iron Concentration (LIC).
干预措施: deferasirox (Drug)
结局指标
主要结局
Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
时间窗: Baseline, 52 weeks
Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment
次要结局
- Absolute Change in LIC From Baseline Over Time(24, 52, 76, 104, 128, 156, 180, 208, 232, 260 Weeks)
- PK Parameters: AUCtau(pre-dose (0 hour), and at 2, and 4 hours at Week 4)
- Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw(5 years)
- Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period(post-baseline, up to 260 weeks)
- Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)(Baseline, End of Study (EOS): Week 260 + 30 days follow up)
- PK Parameters: Tmax(pre-dose (0 hour), and at 2, and 4 hours at Week 4)
- Plasma Pharmacokinetics (PK) Deferasirox Concentrations(Weeks 12 & 24: pre-dose (0hr), 2hr & 4hr post-dose)
- Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)(Week 24, End of Study (EOS): Week 260 + 30 days follow up)
- Absolute Change in Serum Ferritin From Baseline After 52 Weeks(Baseline, 52 weeks)
- Time to Achieving LIC <5 mg Fe/g dw(5 years)
- Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)(Baseline, 52, 104 & 156 Weeks)
- Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)(Baseline, 52, 104 & 156 Weeks)
- Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome(Baseline, 52 Weeks)
- PK Parameters: Cmax(pre-dose (0 hour), and at 2, and 4 hours at Week 4)
研究者
研究点 (1)
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