NCT03993613终止2 期
Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia
Prothya Biosolutions2 个研究点 分布在 1 个国家目标入组 10 人开始时间: 2019年3月21日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Erythropoiesis
研究概览
简要总结
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anaemia in combination with an elevated HbA2 (>2.5%) and a haemoglobin of <6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule.
- •Age above≥ 17 years.
- •Adequate renal and hepatic function tests
- •WHO performance 0, 1 or
- •Signed informed consent.
排除标准
- •Known with allergic reactions against human plasma or plasma products.
- •Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease).
- •Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias.
- •Pregnant or lactating females.
- •Known with IgA deficiency with anti-IgA antibodies
结局指标
主要结局
Erythropoiesis
时间窗: 17 weeks
Change of haemoglobin level and/or or change of number of RBC units transfused/week
次要结局
- Change from baseline in serum iron(17 weeks)
- Change from baseline in change plasma levels of advanced glycation end products(17 weeks)
- Change in spleen size(at baseline and at 16 weeks)
- Change from baseline in reticulocyte count(17 weeks)
- Change from baseline in erythropoietin levels(17 weeks)
- Ctrough(predose)
- Cmin(predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days)
- tmax(predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days)
- Cmax(predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days)
- AUCτ(predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days)
- Adverse events(17 weeks)
研究者
研究点 (2)
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