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NCT Number: NCT07409259

PT-MSCs Exosome Injection in the Treatment of Chronic-to-acute Liver Failure

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.

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Key information

Conditions

Acute-on-Chronic Liver Failure (ACLF) Acute-On-Chronic Liver Failure Digestive System Diseases Hepatic Insufficiency Liver Diseases Liver Failure Liver Failure, Acute

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Third Affiliated Hospital, Sun Yat-sen University Guangzhou, Guangdong, China 510630

Guangzhou, Guangdong, 510630, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 < 7.4.
  • Participants must be fully informed about the study and voluntarily sign a written informed consent form prior to participation.

Exclusion criteria

  • 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.

Treatment and study plan

exosome injection

Biological

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.

standard comprehensive internal medical treatment,

Combination Product

Participants will receive standard comprehensive internal medical treatment, including antiviral therapy, hepatoprotective drugs, and symptomatic support.

Primary outcomes

  1. 12-Week Survival Rate

    Time frame: 12 weeks

    The percentage of participants who are alive at 12 weeks after enrollment.

Secondary outcomes

  1. Changes from the baseline in COSSH-ACLF II score

    Time frame: 12 weeks

    The Chinese Severe Hepatitis B Study Group (COSSH), based on a multicenter, open-label large cohort study, has established a novel prognostic scoring system for hepatitis B virus (HBV)-ACLF: COSSH-ACLF II s. The COSSH-ACLF II score is calculated as: 1.649×ln(INR) + 0.457×HE grade + 0.425×ln(neutrophils) + 0.396×ln(TBil) + 0.576×ln(urea) + 0.033×age. Theoretically, the score ranges from ~1.0 to ~15.0, though clinically observed values typically fall between 4.5 and 10.0. A score of less than 7.4 indicates a low-risk group, a score between 7.4 and 8.4 indicates a moderate-risk group, and a score greater than 8.4 indicates a high risk of 28- and 90-day mortality.Higher scores indicate greater severity of liver failure and a worse outcome.

  2. Changes from the baseline in Child-Pugh score

    Time frame: 12-weeks

    The Child-Pugh Score assesses the prognosis of chronic liver disease based on five indicators: hepatic encephalopathy, ascites, total bilirubin, albumin, and PT/INR. The total score ranges from 5 to 15. Higher scores indicate worse liver function. (Classification: Grade A = 5-6 points; Grade B = 7-9 points; Grade C = 10-15 points.)

  3. Change from Baseline in Clinical Symptom Score

    Time frame: 12 weeks

    Assessment of clinical symptoms including fatigue, anorexia, nausea, jaundice, and level of consciousness. Each symptom is graded on a scale from 0 (absent) to 3(severe). The individual scores are summed to calculate a total symptom score ranging from 0 to 15. Higher scores indicate more severe symptoms.

  4. 4-Week Survival Rate

    Time frame: 4 weeks

    The percentage of participants who are alive at 4 weeks after enrollment

  5. The rate of participants with adverse outcomes at Week 4 and Week 12

    Time frame: 4-week , 12-week

    The rate of adverse outcomes, defined as death, treatment abandonment, or liver transplantation.

Other outcomes

  1. Change from Baseline in Hemoglobin

    Time frame: Day1, Day4、Day7、Day10、Day13、Day28、Day84

    Assessment of hemoglobin concentration. Presented in units of g/L.

  2. Change from Baseline in International Normalized Ratio (INR)

    Time frame: Day1, Day4、Day7、Day10、Day13、Day28、Day84

    Assessment of coagulation function via INR. INR is a standardized ratio without units. Higher values indicate worse coagulation function.

  3. Change from Baseline in Interleukin-6 (IL-6)

    Time frame: Day1, Day4、Day7、Day10、Day13、Day28、Day84

    Measurement of serum Interleukin-6 concentration to evaluate the pro-inflammatory cytokine response. Presented in units of pg/mL.

  4. Change from Baseline in White Blood Cell (WBC) Count

    Time frame: Day1, Day4、Day7、Day10、Day13、Day28、Day84

    Assessment of inflammation through total white blood cell count. Presented in units of 10^9/L.

  5. Number of participants with treatment-related adverse Events (AEs) and serious Adverse Events (SAEs)

    Time frame: 12 weeks

    Safety assessment evaluating the frequency and severity of adverse events throughout the study duration. All treatment-related adverse events will be assessed by CTCAE v5.0.

Sponsors and collaborators

Lead sponsor

Third Affiliated Hospital, Sun Yat-Sen University

Other

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Feb 13, 2026
Registry last updated
Feb 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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