NCT00602446终止2 期
Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Number of Patients Not Completing Treatment
研究概览
简要总结
RATIONALE: Deferasirox may be effective in treating iron overload caused by blood transfusions in patients who have undergone donor stem cell transplant.
PURPOSE: This phase II trial is studying the side effects and how well deferasirox works in treating patients with iron overload after donor stem cell transplant.
详细描述
OBJECTIVES:
Primary
- To evaluate the safety of deferasirox given over 6 months in reducing liver iron concentration in patients with transfusional iron overload after undergoing allogeneic hematopoietic stem cell transplantation.
Secondary
- To evaluate the efficacy of deferasirox in reducing liver iron overload in these patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of iron overload, defined as serum ferritin > 1,000 ng/mL and liver iron concentration ≥ 5 mg iron/g on tissue proton transverse relaxation rates Magnetic Resonance Imaging (MRI)
- •Underwent prior allogeneic hematopoietic stem cell transplantation (HSCT) using either myeloablative or reduced-intensity conditioning at least 12 months ago
- •No evidence of relapse or progression of the primary disease for which allogeneic HSCT was performed
- •Patients who have become red-cell transfusion independent (i.e., no red cell transfusions within the past 3 months) as well as patients who require red cell transfusions are eligible
- •Meets one of the following criteria:
- •Ineligible for phlebotomy (hemoglobin < 11 g/dL, poor intravenous access, or unable to undergo phlebotomy every 4 weeks)
- •Have failed treatment with phlebotomy (serum ferritin > 50% of baseline after 3 months of phlebotomy)
- •Refused phlebotomy
- •ECOG performance status of 0-2
- •Life expectancy ≥ 6 months
- •Adequate renal function defined as serum creatinine < or = 1.6 mg/dL and creatinine clearance of > or = 60 ml/min calculated using the Crockcroft-Gault formula on 2 occasions within 30 days of enrollment
- •Sexually active men and women must use an effective method of contraception. Alternatively, women must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal.
- •Must be able to give written informed consent.
- •Prior therapy with deferoxamine allowed provided it was completed ≥ 12 months ago
排除标准
- •Contraindication for performing MRI or inability to undergo MRI because of claustrophobia or weight (>350 pounds).
- •Inability to take medications orally.
- •Uncontrolled bacterial, viral, or fungal infection
- •ANC ≥ 1,000/mm³
- •Hemoglobin ≥ 8.0 g/dL
- •Platelet count ≥ 50,000/mm³
- •Aspartate aminotransferance (AST) and alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal
- •Less than 4 weeks since prior and no concurrent systemic investigational drug
- •Less than 7 days since prior and no concurrent topical investigational drug. Concurrent non-investigational medications needed to treat concomitant medical conditions are allowed, with the exception of other chelating agents. Concurrent growth factors such as epoetin alfa, darbepoetin alfa, filgrastim (G-CSF), and sargramostim (GM-CSF) allowed. Concurrent irradiated packed red-cell and platelet transfusions allowed as clinically indicated. Concurrent low-doses of vitamin C supplements (≤ 200 mg/day) allowed.
- •Concurrent iron supplements or multivitamins with iron.
- •Aluminum-containing antacid therapies may not be taken simultaneously with deferasirox, but may be taken 2 hours before or after administration of deferasirox
- •On dialysis or status post-renal transplantation
- •Pregnant or nursing
研究组 & 干预措施
Deferasirox Treated
Experimental
Includes patients that were treated with deferasirox for 6 months.
干预措施: deferasirox (Drug)
结局指标
主要结局
Number of Patients Not Completing Treatment
时间窗: 6 Months
Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity
次要结局
- Reduction in Liver Iron Concentration After Study Drug(6 Months)
研究者
研究点 (1)
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