A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 4
- 主要终点
- Improvement rate of Child-Pugh score (Phase 2)
研究概览
简要总结
This is a first-in-human Phase1/2 study of ADR-001, adipose-derived mesenchymal stem cells (AD-MSCs). The safety and preliminary efficacy are evaluated in Phase 1 in patients with liver cirrhosis caused by Hepatitis C or Nonalcoholic Steatohepatitis and a recommended Phase 2 dose is determined by the evaluation. The exploratory efficacy and safety are investigated against the same target population in Phase 2.
详细描述
Patients with decompensated liver cirrhosis (Child-Pugh score; Grade B) caused by Hepatitis C or Nonalcoholic Steatohepatitis are enrolled to the study. In Phase 1, one of 3 doses of AD-MSCs is administered by 1 hour single intravenous infusion. Patients are hospitalized for 1 week and a recommended dose for Phase 2 is determined by the evaluation of the safety and efficacy. In Phase 2, patients with the same disease criteria are enrolled and dosed to investigate the exploratory efficacy and safety.
The safety and efficacy are evaluated until 24 weeks after dosing both in Phase 1 and Phase 2.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and women ≥ 20 years of age
- •Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)
- •Child-Pugh grade B liver cirrhosis
- •ECOG Performance Status ≤ 2
排除标准
- •Liver cirrhosis patients other than hepatitis C or NASH
- •Malignant neoplasm (except hepatocellular carcinoma patients without recurrence more than 2 years)
- •History of venous thrombosis or pulmonary embolism
- •Serum creatinine ≥ 2 mg/dL or T-Bil ≥ 5.0 mg/dL
- •Infection with hepatitis B, HIV, ATLV-1 or parvovirus B19
- •Patients experienced transplantation or cell therapy
- •Pregnancy or positive on pregnancy test
- •Complications of significant heart disease, kidney disorder, or respiratory disease
- •Drug or alcohol abuse
结局指标
主要结局
Improvement rate of Child-Pugh score (Phase 2)
时间窗: 24 weeks
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Safety profile of ADR-001 including the incidence of adverse events (Phase 1)
时间窗: 24 weeks
Safety will be evaluated based on the medical review of adverse event reports and the results of clinical laboratory tests, vital sign, and physical examinations.
次要结局
- Change of liver function evaluated by Child-Pugh score (Phase 2)(24 weeks)
- Change of liver function evaluated by Child-Pugh score (Phase 1)(24 weeks)
- Safety profile of ADR-001 including the incidence of adverse events (Phase 2)(24 weeks)
- Improvement rate of Child-Pugh score (Phase 1)(24 weeks)
- Improvement rate of Child-Pugh grade (Phase 1)(24 weeks)
- Improvement rate of Child-Pugh grade (Phase 2)(24 weeks)