Single-center, Prospective, Randomized Controlled Study of PT-MSCs Exosome Injection (Code: PT-MSCs-EVS-2023-1) in the Treatment of Chronic-to-acute Liver Failure
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- 12-Week Survival Rate
研究概览
简要总结
The goal of this single-center, prospective, randomized controlled study is to evaluate the safety and efficacy of PT-MSCs Exosome Injection (code: PT-MSCs-EVS-2023-1) in treating patients with acute-on-chronic liver failure (ACLF). As an exploratory study with a small sample size, the primary focus is to observe safety outcomes and preliminary efficacy. The study will enroll patients aged 18 to 65 years who meet the diagnostic criteria for acute-on-chronic liver failure as outlined in the 2024 Chinese Liver Failure Clinical Guidelines and have a COSSH-ACLF II score of less than 7.4.
The main questions this study aims to answer are:
Does the combined therapy improve the 12-week survival rate compared to standard treatment alone? What is the safety profile of the PT-MSCs Exosome Injection (incidence of adverse events and serious adverse events)? Does the treatment improve clinical symptoms (such as fatigue, anorexia, and jaundice) and biochemical markers (liver function, coagulation, inflammation)? Does the treatment improve the 4-week survival rate and reduce the incidence of adverse outcomes (death, treatment abandonment, or liver transplantation)?
Participants will be recruited and screened to ensure eligibility. A total of 20 eligible participants will be randomly allocated into one of two groups in a 1:1 ratio using block randomization:
Control Group (10 patients): Participants will receive standard comprehensive internal medical treatment, including antiviral therapy, hepatoprotective drugs, and symptomatic support. Artificial liver support or liver transplantation may be performed based on the investigator's clinical judgment.
Study Group (10 patients): In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10^11 particles per dose).
For the Study Group, the exosome injection is diluted in 100 mL of normal saline and administered via intravenous infusion over a period of not less than 2 hours. Treatment is administered once every 3 days (on Day 1, Day 4, Day 7, and Day 10) for a total of 4 doses. All participants will undergo follow-up assessments at specific intervals (Days 4, 7, 10, 13, 28, and 84) to monitor survival, clinical improvement, and safety signals.
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
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18 to 65 years, regardless of gender.
- •Diagnosed with Acute-on-Chronic Liver Failure (ACLF) according to the diagnostic criteria in the "Guidelines for Diagnosis and Treatment of Liver Failure (2024 Edition)."
- •COSSH-ACLF II score < 7.
- •Participants must be fully informed about the study and voluntarily sign a written informed consent form prior to participation.
排除标准
- •Patients with chronic liver failure.
- •Patients with active bleeding or Disseminated Intravascular Coagulation (DIC) that has not been effectively controlled.
- •Known allergy to blood products or any medications/drugs used in the treatment protocol.
- •Patients with circulatory failure.
- •History of myocardial infarction, cerebral infarction, or cerebral hemorrhage within the past 6 months.
- •History of malignancy within the past 5 years (excluding cured basal cell carcinoma of the skin or carcinoma in situ of the cervix).
- •Pregnant or lactating women.
- •Any other conditions that, in the opinion of the investigator, make the patient unsuitable for participation in the trial.
研究组 & 干预措施
Control Group
Participants will receive standard comprehensive internal medical treatment, including antiviral therapy, hepatoprotective drugs, and symptomatic support. Artificial liver support or liver transplantation may be performed based on the investigator's clinical judgment.
干预措施: standard comprehensive internal medical treatment, (Combination Product)
Study Group
In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10^11 particles per dose).
干预措施: exosome injection (Biological)
Study Group
In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10^11 particles per dose).
干预措施: standard comprehensive internal medical treatment, (Combination Product)
结局指标
主要结局
12-Week Survival Rate
时间窗: 12 weeks
The percentage of participants who are alive at 12 weeks after enrollment.
次要结局
- Changes from the baseline in COSSH-ACLF II score(12 weeks)
- Changes from the baseline in Child-Pugh score(12-weeks)
- Change from Baseline in Clinical Symptom Score(12 weeks)
- 4-Week Survival Rate(4 weeks)
- The rate of participants with adverse outcomes at Week 4 and Week 12(4-week , 12-week)
研究者
Liang Peng
Professor
Third Affiliated Hospital, Sun Yat-Sen University