NCT00110617已完成2 期
A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions
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
- 212
- 试验地点
- 58
- 主要终点
- The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
研究概览
简要总结
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
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
- •Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)
- •Iron overload from repeated blood transfusion, as defined by one of the following:
- •For patients > 16 years old receiving simple transfusions: lifetime history of receipt of at least 120 ml/kg or 30 adult units of packed red blood cells, OR
- •For patients ≤ 16 years old receiving simple transfusions: lifetime history of receipt of at least 120 ml/kg of packed red blood cells, OR
- •For all patients receiving exchange transfusions in the absence of a previous attempt to achieve negative iron balance: lifetime performance of at least 20 procedures, OR
- •For all patients: liver iron content ≥ 7 mg Fe/g dry weight as measured by biopsy, Magnetic Resonance Imaging (MRI), or magnetic susceptibility performed within 3 months prior to entry into screening
- •For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two occasions during the prior year obtained in the absence of concomitant infection.
- •Body weight > 10 kg
- •No known allergy or contraindication to the administration of deferoxamine
- •Ability to comply with all study-related procedures, medications, and evaluations
- •Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
- •Written informed consent by the patient or for pediatric patient's consent of the patient's legal guardian. The definition of the term 'pediatric' for enrollment and study conduct will be in accordance with the local legislation.
排除标准
- •Serum creatinine above the upper limit of normal
- •Significant proteinuria
- •History of nephrotic syndrome
- •Alanine aminotransferase (ALT) ≥ 250 U/L at screening
- •Clinical evidence of active hepatitis B or hepatitis C
- •History of HIV
- •Fever or other signs/symptoms of infection within 10 days prior to the screening visit
- •Uncontrolled systemic hypertension
- •History of Myocardial Infarction, Congestive Heart Failure or unstable cardiac disease not controlled by standard medical therapy
- •Clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation
- •Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
- •History of drug or alcohol abuse within the 12 months prior to enrollment
- •Pregnant or breast feeding patients
- •Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug 7 days prior to the screening visit
- •Randomization in a previous clinical trial involving ICL670
- •Other protocol-related inclusion / exclusion criteria may apply.
研究组 & 干预措施
Deferasirox (ICL670)
Experimental
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
干预措施: Deferasirox (ICL670) (Drug)
Deferoxamine (DFO) then ICL670
Experimental
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
干预措施: Deferasirox (ICL670) (Drug)
Deferoxamine (DFO) then ICL670
Experimental
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
干预措施: Deferoxamine (DFO) (Drug)
结局指标
主要结局
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
时间窗: 24 Weeks
The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.
次要结局
- Absolute Change in Serum Ferritin From Baseline to Week 24(Baseline, 24 Weeks)
- Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52(Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks)
- Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104(Start of Deferasirox (ICL670) treatment, 104 Weeks)
研究者
研究点 (58)
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