Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation (TICATS Study)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Cardiac iron overload
研究概览
简要总结
Hypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia.
Protocol: Sixty subjects with transfusion dependent β-Thalassemia receiving deferasirox iron chelation therapy will be recruited and randomized in a 1:1 ratio to either (1) placebo and continuation of their iron chelation or (2) a combination of iron chelation plus taurine. Transfusion and safety visits will be scheduled monthly with clinical/biochemical assessment visits every three months. The efficacy of taurine combined with standard chelation therapy will be assessed at baseline and 12 months posttreatment by both cardiac T2*MRI, and cardiac function. The recruitment period is projected to be 12 months from initiation.
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
Both taurine and placebo powder inside the capsules are a white crystalline, odorless powder. The powder is loaded into clear, vegetarian, odorless capsules. Placebo and Investigational Product capsules will be packaged in 500 cc HDPE bottles.
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
- •Participant inclusion criteria
- •Participants who meet all of the following criteria will qualify for entry into the study:
- •Diagnosis of transfusion-dependent Thalassemia being followed at the RBC clinic at TGH
- •Age 18 or older
- •On a stable dose of iron chelation for >30 days. Combination iron chelation regimen is allowed in the study
- •Cardiac MRI T2* ≥8ms measured within 3 months prior to randomization.
- •Preserved left ventricular ejection fraction (LVEF) >50% as measured by cardiac MRI measured within 3 months prior to randomization.
- •Participant
排除标准
- •Participants who meet any of the following criteria will be excluded from the study:
- •More than 16 transfusions in the past 12 months or those who are anticipated to be on a 3 week transfusion schedule during the study period
- •Serum ferritin < 500 ng/mL at screening
- •Liver iron concentration > 40 mg/g dw as measured by liver R2 MRI (FerriScan) measured within 3 months prior to randomization
- •Signs and symptoms consistent with congestive heart failure in the opinion of the investigator
- •As a result of medical review, physical examination or screening investigations, the investigator considers the subject unfit for the study.
- •No fixed address
- •Interval advent of general contraindications to MRI.
- •Taking another investigational product within 30 days of anticipated date of randomization
- •Women who are currently pregnant or plan to become pregnant during the study period
研究组 & 干预措施
Placebo
placebo four times daily
干预措施: Placebo (Other)
Taurine
675mg taurine four times daily
干预措施: Taurine (Dietary Supplement)
结局指标
主要结局
Cardiac iron overload
时间窗: 12 months
Differences in ratio (T2\* at year 1)/(T2\* at baseline) between the taurine and standard chelation therapy arm
次要结局
- C-reactive protein(12 months)
- Reduced glutathione(12 months)
- Left ventricular ejection fraction(12 months)
- Blood taurine level(12 months)
- Plasma MDA(12 months)
- Interleukin-6(12 months)
研究者
Kevin H.M. Kuo, MD, MSc, FRCPC
Principal Investigator
University Health Network, Toronto