Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
- 试验地点
- 4
- 主要终点
- Hemoglobin
研究概览
简要总结
Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to investigate the pharmacokinetics, efficacy and safety of Apotransferrin replacement therapy in atransferrinemia patients.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl
- •Informed consent
排除标准
- •Known with allergic reactions against human plasma or plasma products
- •Having detectable anti-immunoglobulin A antibodies
研究组 & 干预措施
human apotransferrin
intravenous apotransferrin every 4-8 weeks
干预措施: Human apotransferrin (Drug)
结局指标
主要结局
Hemoglobin
时间窗: 15 year
Hemoglobin within normal range
pharmacokinetics of transferrin
时间窗: first infusion and year 3
serum transferrin levels measured on several time points before and after infusion
iron overload in organs
时间窗: 15 years
Elimination of iron overload (liver, heart)
pharmacokinetics of transferrin
时间窗: first infusion and year 3
serum transferrin levels measured on several time points before and after infusion
次要结局
- Erythrocytes(15 years)
- Iron overload(15 years)
- Hematocrit(15 years)