Acute-on-Chronic Liver Failure (ACLF)Early Phase 1NCT07409259
PT-MSCs Exosome Injection in the Treatment of Chronic-to-acute Liver Failure
A Early Phase 1 study testing exosome injection and standard comprehensive internal medical treatment, in people with Acute-on-Chronic Liver Failure (ACLF), run by Third Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute-on-Chronic Liver Failure (ACLF)
- What is being tested?
- exosome injection and standard comprehensive internal medical treatment,
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2026to 31 Aug 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Third Affiliated Hospital, Sun Yat-sen University Guangzhou, Guangdong, China 510630
Guangzhou, Guangdong, 510630
About this study
Official 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
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:
Read more
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)
- Being tested
- exosome injection, standard comprehensive internal medical treatment,
- Keywords
- Acute -On-Chronic Liver Failure, Bio-artificial liver, Exosome, Mesenchymal stem cells
- Medical subject headings
- Acute-On-Chronic Liver Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
exosome injectionBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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). Receives: Biological: exosome injection; Combination Product: standard comprehensive internal medical treatment, |
What is being measured
The primary outcome is the main question the study is designed to answer.
12-Week Survival Rate
The percentage of participants who are alive at 12 weeks after enrollment.
Measured: 12 weeks
Secondary outcomes (5)
Changes from the baseline in COSSH-ACLF II score
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.
Measured: 12 weeks
Changes from the baseline in Child-Pugh score
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.)
Measured: 12-weeks
Change from Baseline in Clinical Symptom Score
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.
Measured: 12 weeks
4-Week Survival Rate
The percentage of participants who are alive at 4 weeks after enrollment
Measured: 4 weeks
The rate of participants with adverse outcomes at Week 4 and Week 12
The rate of adverse outcomes, defined as death, treatment abandonment, or liver transplantation.
Measured: 4-week , 12-week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2026
- Study start1 Jan 2026
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute-on-Chronic Liver Failure (ACLF)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Third Affiliated Hospital, Sun Yat-Sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Liang Peng, Third Affiliated Hospital, Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07409259
- Sponsor's protocol ID
- PL21
- Status last verified
- Jan 2026
- First submitted
- 12 Jan 2026
- Last updated
- 13 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07409259
- Acronym / sponsor ID
- PL21
- Conditions
- Acute-on-Chronic Liver Failure (ACLF)
- Keywords
- Acute -On-Chronic Liver Failure, Bio-artificial liver, Exosome, Mesenchymal stem cells
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Third Affiliated Hospital, Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- 1 Jan 2026 (actual)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 13 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control Group | Active comparator | Combination Product: standard comprehensive internal medical treatment,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 | Experimental | Biological: exosome injection, Combination Product: standard comprehensive internal medical treatment,In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10^11 particles per dose). |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| exosome injectionFor 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. | Biological | Study Group |
| standard comprehensive internal medical treatment,Participants will receive standard comprehensive internal medical treatment, including antiviral therapy, hepatoprotective drugs, and symptomatic support. | Combination product | Control Group, Study Group |
Primary outcome
12-Week Survival Rate
Time frame: 12 weeks
The percentage of participants who are alive at 12 weeks after enrollment.
5 secondary outcomes
Changes from the baseline in COSSH-ACLF II score
Time frame: 12 weeks
Changes from the baseline in Child-Pugh score
Time frame: 12-weeks
Change from Baseline in Clinical Symptom Score
Time frame: 12 weeks
4-Week Survival Rate
Time frame: 4 weeks
The rate of participants with adverse outcomes at Week 4 and Week 12
Time frame: 4-week , 12-week
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing exosome injection and standard comprehensive internal medical treatment, in people with Acute-on-Chronic Liver Failure (ACLF), run by Third Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
12-Week Survival Rate (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Feb 2026.