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

NCT07409259 | Active Not Recruiting Early Phase 1 DMC

• 1 other identifier: PL21
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Acute-on-Chronic Liver Failure (ACLF)

Keywords

Acute -On-Chronic Liver Failure; Bio-artificial liver; Exosome; Mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

• 12-Week Survival Rate

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

  12 weeks

Secondary Outcomes (5)

• 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

Other Outcomes (5)

• Change from Baseline in Hemoglobin

  Day1, Day4、Day7、Day10、Day13、Day28、Day84

• Change from Baseline in International Normalized Ratio (INR)

  Day1, Day4、Day7、Day10、Day13、Day28、Day84

• Change from Baseline in Interleukin-6 (IL-6)

  Day1, Day4、Day7、Day10、Day13、Day28、Day84

Study Arms (2)

Control Group

ACTIVE COMPARATOR

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.

Combination Product: standard comprehensive internal medical treatment,

Study Group

EXPERIMENTAL

In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10\^11 particles per dose).

Biological: exosome injection; Combination Product: standard comprehensive internal medical treatment,

Interventions

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.

Study Group

standard comprehensive internal medical treatment, COMBINATION_PRODUCT

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

Control Group; Study Group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Third Affiliated Hospital, Sun Yat-Sen University: lead

Study Sites (1)

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

Guangzhou, Guangdong, 510630, China

Location

MeSH Terms

Conditions

Acute-On-Chronic Liver Failure

Condition Hierarchy (Ancestors)

Liver Failure, Acute; Liver Failure; Hepatic Insufficiency; Liver Diseases; Digestive System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: January 12, 2026
• First Posted: February 13, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2027
• Last Updated: February 13, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)