Ignite Creation Date:
2026-03-26 @ 3:14 PM
Ignite Modification Date:
2026-03-31 @ 4:12 AM
Study NCT ID:
NCT07409259
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2026-02-13
First Post:
2026-01-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
PT-MSCs Exosome Injection in the Treatment of Chronic-to-acute Liver Failure
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D065290', 'term': 'Acute-On-Chronic Liver Failure'}], 'ancestors': [{'id': 'D017114', 'term': 'Liver Failure, Acute'}, {'id': 'D017093', 'term': 'Liver Failure'}, {'id': 'D048550', 'term': 'Hepatic Insufficiency'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2026-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-01', 'completionDateStruct': {'date': '2027-08-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-02-06', 'studyFirstSubmitDate': '2026-01-12', 'studyFirstSubmitQcDate': '2026-02-06', 'lastUpdatePostDateStruct': {'date': '2026-02-13', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-08-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change from Baseline in Hemoglobin', 'timeFrame': 'Day1, Day4、Day7、Day10、Day13、Day28、Day84', 'description': 'Assessment of hemoglobin concentration. Presented in units of g/L.'}, {'measure': 'Change from Baseline in International Normalized Ratio (INR)', 'timeFrame': 'Day1, Day4、Day7、Day10、Day13、Day28、Day84', 'description': 'Assessment of coagulation function via INR. INR is a standardized ratio without units. Higher values indicate worse coagulation function.'}, {'measure': 'Change from Baseline in Interleukin-6 (IL-6)', 'timeFrame': 'Day1, Day4、Day7、Day10、Day13、Day28、Day84', 'description': 'Measurement of serum Interleukin-6 concentration to evaluate the pro-inflammatory cytokine response. Presented in units of pg/mL.'}, {'measure': 'Change from Baseline in White Blood Cell (WBC) Count', 'timeFrame': 'Day1, Day4、Day7、Day10、Day13、Day28、Day84', 'description': 'Assessment of inflammation through total white blood cell count. Presented in units of 10\\^9/L.'}, {'measure': 'Number of participants with treatment-related adverse Events (AEs) and serious Adverse Events (SAEs)', 'timeFrame': '12 weeks', 'description': '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.'}], 'primaryOutcomes': [{'measure': '12-Week Survival Rate', 'timeFrame': '12 weeks', 'description': 'The percentage of participants who are alive at 12 weeks after enrollment.'}], 'secondaryOutcomes': [{'measure': 'Changes from the baseline in COSSH-ACLF II score', 'timeFrame': '12 weeks', 'description': '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.'}, {'measure': 'Changes from the baseline in Child-Pugh score', 'timeFrame': '12-weeks', 'description': '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.)'}, {'measure': 'Change from Baseline in Clinical Symptom Score', 'timeFrame': '12 weeks', 'description': '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.'}, {'measure': '4-Week Survival Rate', 'timeFrame': '4 weeks', 'description': 'The percentage of participants who are alive at 4 weeks after enrollment'}, {'measure': 'The rate of participants with adverse outcomes at Week 4 and Week 12', 'timeFrame': '4-week , 12-week', 'description': 'The rate of adverse outcomes, defined as death, treatment abandonment, or liver transplantation.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Acute -On-Chronic Liver Failure', 'Bio-artificial liver', 'Exosome', 'Mesenchymal stem cells'], 'conditions': ['Acute-on-Chronic Liver Failure (ACLF)']}, 'descriptionModule': {'briefSummary': "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.\n\nThe main questions this study aims to answer are:\n\nDoes 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)?\n\nParticipants 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:\n\nControl 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.\n\nStudy 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).\n\nFor 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Aged 18 to 65 years, regardless of gender.\n* 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)."\n* COSSH-ACLF II score \\< 7.4.\n* Participants must be fully informed about the study and voluntarily sign a written informed consent form prior to participation.\n\nExclusion Criteria:\n\n* Patients with chronic liver failure.\n* Patients with active bleeding or Disseminated Intravascular Coagulation (DIC) that has not been effectively controlled.\n* Known allergy to blood products or any medications/drugs used in the treatment protocol.\n* Patients with circulatory failure.\n* History of myocardial infarction, cerebral infarction, or cerebral hemorrhage within the past 6 months.\n* History of malignancy within the past 5 years (excluding cured basal cell carcinoma of the skin or carcinoma in situ of the cervix).\n* Pregnant or lactating women.\n* Any other conditions that, in the opinion of the investigator, make the patient unsuitable for participation in the trial.'}, 'identificationModule': {'nctId': 'NCT07409259', 'briefTitle': 'PT-MSCs Exosome Injection in the Treatment of Chronic-to-acute Liver Failure', 'organization': {'class': 'OTHER', 'fullName': 'Third Affiliated Hospital, Sun Yat-Sen University'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'PL21'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Control Group', 'description': "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.", 'interventionNames': ['Combination Product: standard comprehensive internal medical treatment,']}, {'type': 'EXPERIMENTAL', 'label': 'Study Group', 'description': 'In addition to the standard treatment received by the control group, participants will receive PT-MSCs Exosomes (2×10\\^11 particles per dose).', 'interventionNames': ['Biological: exosome injection', 'Combination Product: standard comprehensive internal medical treatment,']}], 'interventions': [{'name': 'exosome injection', 'type': 'BIOLOGICAL', 'description': '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.', 'armGroupLabels': ['Study Group']}, {'name': 'standard comprehensive internal medical treatment,', 'type': 'COMBINATION_PRODUCT', 'description': 'Participants will receive standard comprehensive internal medical treatment, including antiviral therapy, hepatoprotective drugs, and symptomatic support.', 'armGroupLabels': ['Control Group', 'Study Group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '510630', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'facility': 'Third Affiliated Hospital, Sun Yat-sen University Guangzhou, Guangdong, China 510630', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Third Affiliated Hospital, Sun Yat-Sen University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Liang Peng', 'investigatorAffiliation': 'Third Affiliated Hospital, Sun Yat-Sen University'}}}}