Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 213
- 试验地点
- 1
- 主要终点
- difference between multiple observations of the serum ferritin concentrations
研究概览
简要总结
Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published.
This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.
详细描述
The trial was designed as a multicentre randomised open-label trial with blinded data management and data analyses, to assess whether either treatment was superior to the other. The trial was performed on behalf of the Italian Society for the study of Thalassemia and Haemoglobinopathies (SoSTE). The investigators initiated, carried out, and controlled the trial, which was conducted without influence of the non-commercial sponsor.16
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 13 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age
排除标准
- •Known intolerance to one of the trial treatments
- •Platelet count < 100,000/mm3 or or leukocyte count < 3,000/mm3
- •Severe liver damage indicated by ascites
- •Heart failure
研究组 & 干预措施
2
Deferiprone alone at 75 mg/kg divided into three oral daily doses
干预措施: Deferiprone (DFP) (Drug)
1
Sequential treatment including DFP at 75 mg/kg, divided into three oral daily doses, for four days per week and DFO by subcutaneous infusions (8-12h) at 50 mg/kg/day for the remaining three days per week
干预措施: Deferiprone (DFP) and Deferoxamine (DFO) (Drug)
结局指标
主要结局
difference between multiple observations of the serum ferritin concentrations
时间窗: five-year treatment
次要结局
- the difference between the T2* signal at magnetic resonance imaging (MRI) of heart and liver at T0 and T1 time (see below); survival analysis; adverse events; treatment failures; and costs.(five years)