An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Novartis
- 入组人数
- 30
- 试验地点
- 3
- 主要终点
- Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2*
研究概览
简要总结
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
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
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female β-thalassemia outpatients on chronic transfusion therapy (defined as > 8 transfusions per year)
- •Lifetime minimum of 100 previous packed red blood cell transfusions
- •Patients currently on chelation therapy will require a one day wash out prior to the first dose of study drug
- •Age ≥ 10 years
- •Sexually active females of childbearing potential must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy.
排除标准
- •Ejection Fraction < 56 % measured using steady-state free precession imaging by MRI
- •Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes, intrauterine device or any other MRI incompatible metal implants or intractable claustrophobia
- •Abnormal laboratory values as defined by the protocol
- •Clinical or laboratory evidence of active Hepatitis B or Hepatitis C
- •History of HIV positive test result (ELISA or Western blot)
- •Uncontrolled systemic hypertension
- •Second or third degree A-V block
- •Life-threatening arrhythmias, including sustained ventricular tachycardia and aborted sudden death, within the last year
- •History of cardiac conditions or unstable cardiac disease not controlled by standard medical therapy
- •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 (documented negative pregnancy test required for study entry)
- •Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be withdrawn in order to participate in this study
- •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 any drug
- •History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
- •Other inclusion/exclusion criteria may apply
研究组 & 干预措施
deferasirox every day for 77 weeks
Participants received Deferasirox 30 milligrams per kilogram per day (mg/kg/day) orally once daily (OD), 30 minutes before breakfast, preferably around the same time every morning if possible. Deferasirox tablets were dropped into water or orange juice, or apple juice and stirred until completely dispersed. For doses less than 1 gram (g), tablets were dissolved in at least 100 milliliter (mL) of liquid; for doses of 1 to 3 g, tablets were dissolved in at least 200 mL. After tablets were fully disintegrated, the liquid was promptly consumed.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2*
时间窗: From Baseline to 25, 49, 77 Week
Cardiac T2\* was measured in the short axis plane at the widest point of a 4-chamber localizer using custom breath-hold R2\* gradient echo sequences modeled after techniques used by Anderson et al (2001) and Westwood et al (2003).
次要结局
- Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks(From Baseline to 25, 49, 77 Week)
- Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks(From Baseline to 25, 49, 77 Week)
- Serum Ferritin and Changes From Baseline in Serum Ferritin During Study(From Baseline to 25, 49, 77 Week)