An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-risk and INT-1, Myelodysplastic Patients Using Serum Ferritin Monitoring
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 176
- 试验地点
- 36
- 主要终点
- Number of Participants Reporting Adverse Events
研究概览
简要总结
The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
详细描述
Study entry requires a diagnosis of low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria and serum ferritin ≥ 1000 ng/mL. Patients must have had at least 30 prior red blood cell transfusions. Deferasirox will be administered at an initial dose of 20 mg/kg orally once per day. Patient transfusion history and at least three complete blood count (CBC) values must be available for the 12 weeks prior to study registration for patients with MDS and chronic iron overload from blood transfusions.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with low or intermediate (INT-1) risk MDS
- •Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO).
- •Age greater than or equal to 18 years
- •Availability of transfusion records for the 12 weeks prior to registration
- •A lifetime minimum of 30 previous packed red blood cell transfusions
- •Availability of at least three CBC values (pretransfusion) during the 12 weeks prior to registration
- •Serum Ferritin:
- •For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year.
- •Serum ferritin ≥ 1000 ng/mL at screening via the central lab.
- •Life expectancy ≥ 6 months
- •Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
- •Able to provide written informed consent
排除标准
- •Serum creatinine above the upper limit of normal
- •Alanine aminotransferase (ALT) > 500 U/L during screening
- •Clinical or laboratory evidence of active Hepatitis B or C
- •Urinary protein/creatinine ratio > 0.5 mg/mg
- •History of HIV positive test result (ELISA or Western blot)
- •Eastern Cooperative Oncology Group (ECOG) Performance Status > 2
- •Patients with uncontrolled systemic hypertension
- •Unstable cardiac disease not controlled by standard medical therapy
- •Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
- •Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
- •Pregnancy or breast feeding
- •Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
- •Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
- •History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
研究组 & 干预措施
ICL670
Evaluate the safety and tolerability of deferasirox 20 mg/kg/day over one year in patients with MDS
干预措施: Deferasirox (Drug)
结局指标
主要结局
Number of Participants Reporting Adverse Events
时间窗: up to 53 Weeks
Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the study, whether or not considered related to the participant's participation in the study. Serious Adverse Events include adverse events that result in either of death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect.
次要结局
- Change in Serum Ferritin From Baseline to Weeks 13, 25, 37 and 53(From Baseline to Weeks 13, 25, 37 and 53)
- Treatment Compliance to Deferasirox(up to 1 year)
- The Prevalence of Hereditary Hemochromatosis Gene (HFE) Gene Mutations(up to Week 13 (Month 3))
- Change in Labile Plasma Iron (LPI)(From Baseline to Weeks 13, 25, 37 and 49)
- Total Iron Levels(From Baseline to Weeks 13, 25, 37, 49 and 53)
- Directly Chelatable Iron (DCI)(From Baseline to Weeks 13, 25, 37 and 49)
- Transfusion Requirements(up to 1 year)
- Serum Transferrin Levels(From Baseline to Weeks 13, 25, 37, 49 and 53)
- Transferrin Saturation(From Baseline to Weeks 13, 25, 37, 49 and 53)
- Frequency of Hematologic Improvement During the Study(up to 1 year)
- Trough Plasma Deferasirox Concentration(At Week 13, 25, 37 and 49)