A Multicenter, Open-label, Single Arm, Interventional Phase IV Study, to Evaluate the Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 主要终点
- Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
研究概览
简要总结
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the effect of deferasirox therapy on glucose metabolism/homeostasis. Other endocrine parameters complementary or supportive to the primary objective will be assessed and analyzed during this study. A number of lab parameters related to other axes of the endocrine system will be collected and analyzed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Beta thalassemia major and severe intermedia patients transfusion dependent and with transfusional iron overload
- •Patients with diagnosis of impaired fasting glucose or impaired glucose tolerance 4.Patients naïve to deferasirox or patients who already receive deferasirox at sub-optimal doses 5.Cardiac MRI T2* >10 msec; 7.normal cardiac function (LVEF > 56%);
排除标准
- •Non transfusional hemosiderosis;
- •Patients with diabetes mellitus (genetic or secondary) or history of diabetes mellitus in 1st degree relatives;
- •4.Patients who received organ transplant; 5.Patients with galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption; 6.Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox; 7.History of hypersensitivity to the study drug or any of its excipients;
- •Renal impairment
- •Liver impairment; 11.Patients with active chronic hepatitis B infection, active hepatitis C infection;
- •Other protocol-defined inclusion/exclusion criteria may apply" at the end
研究组 & 干预措施
deferasirox
single arm. all patients will receive deferasirox
干预措施: deferasirox (Drug)
结局指标
主要结局
Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
时间窗: 36 months
The primary efficacy variable is the change (mg/dl) from baseline to 36 months of glucose plasma levels measured 2 hr post glusose equivalent oral challange. After a 12-hour overnight fasting, at zero time (baseline) blood sample will be drawn and afterwards patients will receive a glucose-equivalent oral challenge (75 grams). After glucose loading plasma samples will be drawn at 30, 60, 90, 120 minutes for determination of plasma glucose. This will be repeated every 6 month till end of study
次要结局
- Iron overload status(baseline and regularly till end of study (monthly or yearly as specified))
- Safety of deferasirox therapy(baseline and at every scheduled visit (weekly for the first months or after dose escalation and monthly thereafter or yearly till EOS)
- Risk factors for the impairment of glucose homeostasis(baseline and monthly till End of Study)
- change on insulin secretion and sensitivity(baseline and every 6 months measurement of 2hr Glucose OGTT)
- Measurement of thyroid hormones TSH and FT4(baseline and every 12 months)
- Changes in endocrine funcionts parameters(baseline and monthly till EOS)
- Glucose of OGTT ( AUC)(baseline and every 6 months measurement of 2hour Glocose of OGTT)
- Changes in parameters of bone metabolism(baseline, monthly or every 6 months till end of study)