A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Participants With Adverse Events by Primary System Organ Class (SOC)
研究概览
简要总结
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of the planned 12-month core trial, (NCT00390858).
- •Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
- •Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation.
排除标准
- •Pregnant or breast feeding patients
- •Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.
- •Other protocol-defined exclusion criteria may apply.
研究组 & 干预措施
Deferasirox
Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Participants With Adverse Events by Primary System Organ Class (SOC)
时间窗: 4 year extension + core 1 year
Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product.
Change in Liver Iron Concentration (LIC)
时间窗: Baseline of Core Study to End of Extension Study, up to 5 years.
Change in Liver Iron Concentration \[LIC\] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw)
次要结局
- Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)(Baseline of Core Study to End of Extension Study, up to 5 years)
- Relative Change in Serum Ferritin Level(Baseline of Core Study to Extension 18 months, up to 2.5 years.)