NCT01044186已完成2 期
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 9
- 主要终点
- To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity.
研究概览
简要总结
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous. Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study. In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity.
- •Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy.
- •Serum ferritin ≥ 8000 μg/L.
- •Serum ferritin < 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight.
- •Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at > 9 g/dL.
- •Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation.
- •Written, voluntary informed consent.
排除标准
- •Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
- •Patients with non-transfusional hemosiderosis.
- •Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
- •Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
- •Patients with a history of nephrotic syndrome.
- •Patients with a diagnosis of clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation therapy.
- •Patients with severe systemic diseases unrelated to iron overload and which would prevent them from undergoing treatment with ICL
- •Patients with psychiatric or addictive disorders which prevent them from giving informed consent or undergoing treatment with ICL
- •Pregnant or breast feeding patients.
- •Patients treated with systemic investigational drugs within the past four weeks or topical investigational drugs within the past seven days.
- •Any surgical or medical condition which might significantly alter the absorption or excretion of drugs as shown by evidence of any of the following:
- •History of inflammatory bowel disease
- •History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- •History of pancreatic injury or pancreatitis; indication of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- •Patients being considered by the investigator as potentially unreliable and/or not cooperative with regard to the protocol.
- •History of drug or alcohol abuse within the 12 months prior to dosing.
研究组 & 干预措施
ICL670
Experimental
干预措施: ICL670 (Drug)
结局指标
主要结局
To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity.
时间窗: 0 - 163 weeks
次要结局
- To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers.(Yearly)
- To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients.(Quarterly)
- To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron.(Yearly)
研究者
研究点 (9)
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