NCT01254227已完成2 期
Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion (HYPERION)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Change in Cardiac Iron Content From Baseline to Month 12
研究概览
简要总结
This study will evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed be deferasirox monotherapy in patients with severe iron overload due to chronic blood transfusions.
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
- •Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy
- •Myocardial T2* value that is ≥ 5 and < 10 ms
- •Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (MRI)
- •Liver Iron Concentration (LIC) ≥ 7 mg Fe /g dw as determined by R2 MRI.
- •Lifetime history of at least 50 units of red blood cell transfusions, and must be receiving at least ≥ 8 units/yr of red blood cell transfusions
- •Serum ferritin ≥ 1000 ng/mL
排除标准
- •Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
- •Patients unable to undergo study assessments including MRI
- •Patients with serum creatinine greater than Upper limit of normal ULN)range or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥1.0 mg/mg in a non-first void urine sample at baseline.
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Deferasirox
Experimental
Deferoxamine combination followed by Deferasirox monotherapy
干预措施: Deferasirox and Deferoxamine (Drug)
结局指标
主要结局
Change in Cardiac Iron Content From Baseline to Month 12
时间窗: From Baseline to Month 12
Cardiac T2\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\* value of \<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\* or do not have any post-baseline T2\* are excluded from the analysis.
次要结局
- Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24(From the Baseline, Month 6, 12, 18 and Month 24)
- Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24(From the Months 6, 12, 18 and 24)
- Time to Achieve From Baseline (FAS) of at Least 10% at Month 24(At 24 months)
- Change in Cardiac Iron Content From Baseline to Month 6,18 and 24(From Baseline to Months 6, 18 and 24)
- Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24(From the Months 6, 12, 18 and 24)
- Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24(From the Months 6, 12, 18 and 24)
研究者
研究点 (1)
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