NCT00879242已完成2 期
Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec
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
- 20
- 试验地点
- 1
- 主要终点
- Change from baseline in cardiac T2* value
研究概览
简要总结
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
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
- •β-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events
- •Patients naïve for deferasirox
- •Serum ferritin levels ≥1000ng/ml (average of the last 6 months assays);
- •Cardiac MRI T2* >5 and <20 msec.
- •LVEF at MRI ≥56%
排除标准
- •Patients that have already started deferasirox therapy
- •Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.)
- •Clinical conditions requiring intensive chelating therapy on the basis of Investigator's judgment
- •Stable average ALT levels >300 U/L in the preceding 12 months
- •Uncontrolled systemic hypertension
- •Estimated creatinine clearance <60 ml/min
- •History of nephrotic syndrome
- •History of clinically significant ocular toxicity related to the chelating therapy
- •Psychiatric disorders or drug dependence that might impair the capability of the patient to provide their Informed Consent or to be administered the study treatment
- •Known sensitivity to study drug(s) or class of study drug(s)
- •Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study
- •Use of any other investigational agent in the last 30 days.
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Deferasirox
Experimental
30 mg/kg/day. The daily dose can be increased to 40 mg/kg in case of unsatisfactory response after 12 weeks of treatment.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Change from baseline in cardiac T2* value
时间窗: 52 weeks
Unsatisfactory response is a monthly MRI T2\* improvement lower than 3% versus baseline.
次要结局
- Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)(52 weeks)
- Change from baseline liver T2* value(52 weeks)
研究者
研究点 (1)
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