NCT00067080已完成2 期
A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis
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
- 195
- 试验地点
- 34
- 主要终点
- Evaluate the safety and tolerability of multiple doses of ICL670
研究概览
简要总结
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
详细描述
Patients who require repeated blood transfusions accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age greater than or equal to 2 years
- •Sickle cell disease patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- •Serum ferritin greater than 1000 mg/ml
- •Liver iron content greater than 2 mg iron/g dw assessed by means of superconducting quantum interference device (SQUID) for patients who receive simple transfusions and greater than 5 mg iron/ g dw for patients who receive exchange transfusions or who have a history of intermittent blood transfusion.
- •Regular transfusion aimed at maintaining % Hb A above 50% or a previous history of simple transfusion being the recipient of at least 20 units of packed red blood cells.
排除标准
- •Chronic anemias other than sickle cell disease
- •Documented toxicity to deferoxamine
- •Elevated liver enzymes in the year preceeding enrollment
- •Active hepatitis B or hepatitis C
- •HIV seropositivity
- •Elevated serum creatinine or significant proteinuria
- •History of nephrotic syndrome
- •Uncontrolled systemic hypertension
- •Fever and other signs/symptoms of infection within 10 days prior to the start of the study
- •Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
- •Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval (other than beta-adrenergic receptor blocking agents).
- •Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from undergoing any of the treatment options
- •Psychiatric or addictive disorders that would prevent the patient from giving informed consent
- •History of drug or alcohol abuse within the 12 months prior to the study
- •Pregnant or breast feeding patients
- •Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of the study
- •Patients who require concomitant therapy with hydroxyurea
- •Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function
- •Non-compliant or unreliable patients
- •Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography
- •Patients unable to undergo SQUID examination
研究组 & 干预措施
ICL670 + deferoxamine
Experimental
干预措施: ICL670, deferoxamine (Drug)
结局指标
主要结局
Evaluate the safety and tolerability of multiple doses of ICL670
时间窗: 1 year
次要结局
- Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE)(at baseline, after 24 weeks and at 1year (end of study))
- Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables(at 24 and 52 weks pre-dose)
- Evaluate the pharmacokinetics(24 hours post-dose @ 4, 12, 24 and 52 weeks)
- Evaluate the relationship between hepatic iron and potential surrogate markers(at screen, at washout, then every 2 weeks for the first 12 weeks followed by every 4 weeks)
研究者
研究点 (34)
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