The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (NASH) - a Prospective Open Label Phase I/II Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 试验地点
- 9
- 主要终点
- Safety and tolerability of deferasirox in all patients (Phase I)
研究概览
简要总结
This is a Phase I/II open-label uncontrolled, prospective study to assess the clinical and biological effects of Deferasirox (ICL 670, Exjade®) in patients with NASH and increased iron storage / distribution of iron on liver function and liver histology.
NASH is defined clinically and histologically by elevated liver enzymes, signs of hepatic steatosis on ultrasound and magnetic resonance imaging, impaired liver function as expressed by functional breath tests, and significantly altered liver histology.
Patients will be treated in a phase I and phase II part for either 12 or 48 weeks.
Both study parts have different endpoints: in phase I the side effect profile will be evaluated while in phase II the therapeutic response will be tested. Accordingly, measures will be different.
Approximately 10 patients in phase I and 50 patients in phase II will be enrolled according to sample size calculations.
The design is an "adaptive" Two-stage design, allowing to minimize the number of patients included into the trial as well as to introduce corrections for the second stage.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Exjade
干预措施: Exjade (Drug)
结局指标
主要结局
Safety and tolerability of deferasirox in all patients (Phase I)
时间窗: Phase I: 12 weeks of treatment
Safety and tolerability assessments will consist of evaluating (serious) adverse events, laboratory parameters including hematology, chemistry; vital signs and physical examinations according to CTC.
Changes in liver histology in all patients (Phase II)
时间窗: Phase II: 48 weeks of treatment
A decrease in the NASH activity score (NAS) of ≥1 compared to baseline will be classified as response, an unchanged score or an increase in NAS will be judged as non-response.
次要结局
- Phase I: e.g. changes in liver enzymes, serum ferritin, and hemoglobin levels(Phase I: 12 weeks of treatment)
- Phase II: e.g. changes in MRI and histology based assessment of hepatic steatosis, fibrosis and iron content(Phase II: 48 weeks of treatment)