NCT05940610

The Safety and Efficacy of MSC-EVs in Acute/Acute-on-Chronic Liver Failure

Withdrawn Phase 1, PHASE2 Last updated 12 October 2023

What this trial tests

Phase 1, PHASE2 trial testing MSC-EVs in Acute-On-Chronic Liver Failure. Withdrawn.

Timeline

1 September 2023

Primary endpoint
30 September 2024

1 October 2025

Quick facts

Lead sponsor	Third Affiliated Hospital, Sun Yat-Sen University
Phase	Phase 1, PHASE2
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Start date	1 September 2023
Primary completion	30 September 2024
Estimated completion	1 October 2025
Sites	1 location across China

Drugs / interventions tested

• MSC-EVs — full drug profile →

Conditions studied

• Acute-On-Chronic Liver Failure — all drugs for Acute-On-Chronic Liver Failure →
• Acute Liver Failure — all drugs for Acute Liver Failure →

Sponsor

Third Affiliated Hospital, Sun Yat-Sen University

Who can join

Adults 18 to 65, any sex, with Acute-On-Chronic Liver Failure or Acute Liver Failure. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Acute-on-chronic liver failure (ACLF) refers to a liver failure syndrome in which some patients with chronic liver disease with relatively stable liver function suffer from acute liver decompensation and liver failure due to the effects of various acute injury factors，while acute liver failure (ALF) refers to a potentially reversible disorder that was the result of severe liver injury, with an onset of encephalopathy within 8 weeks of symptom appearance and in the absence of pre-existing liver disease. Liver transplantation is the only curative treatment for this type of end-stage liver disease, but the rapid disease progression and lack of donors limit its application. The potential of MSCs to repair or regenerate damaged tissue and suppress immune responses makes them promising in the treatment of liver diseases, especially in the field of liver transplantation. Many studies have shown that MSC-based therapies can reduce the symptoms of liver disease due to their paracrine effects. It has been confirmed in previous studies that infusion of allogeneic MSCs is safe and convenient for patients with ACLF and improve liver function and decrease the incidence of severe infections. Compared to the cells they derive from, mesenchymal stem cells-derived extracellular vesicles (MSC-EVs) are gradually gaining attention for their enhanced safety, as they do not replicate or cause microvascular embolism, and can be easily stored without losing their properties. It represents a novel and effective cell-free therapeutic agent as alternative to cell-based therapies for liver diseases, and liver failure was also concerned. This study was designed to evaluate the safety and efficacy of MSC-EVs in ACLF/ALF .

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Defining tropism and activity of natural and engineered extracellular vesicles.
   Choi W, Park DJ, Eliceiri BP. · · 2024 · cited 18× · PMID 38660297 · DOI 10.3389/fimmu.2024.1363185
2. Mesenchymal stem cell therapy as a game-changer in liver diseases: review of current clinical trials.
   Huang WC, Li YC, Chen PX, Ma KS, et al · · 2025 · cited 14× · PMID 39762946 · DOI 10.1186/s13287-024-04127-y
3. Extracellular vesicle-mediated approaches for the diagnosis and therapy of MASLD: current advances and future prospective.
   Gurjar S, Bhat A R, Upadhya R, Shenoy RP. · · 2025 · cited 10× · PMID 39773634 · DOI 10.1186/s12944-024-02396-3
4. Mesenchymal Stem Cell-Derived Exosomes: Emerging as a Promising Cell-Free Therapeutic Strategy for Autoimmune Hepatitis.
   Wu L, Zhang L, Huang M, Wu Y, et al · · 2024 · cited 9× · PMID 39595530 · DOI 10.3390/biom14111353
5. Therapeutic Potential of Stem Cell-Derived Extracellular Vesicles in Liver Injury.
   Dong J, Luo Y, Gao Y. · · 2024 · cited 2× · PMID 39595055 · DOI 10.3390/biomedicines12112489
6. Rethinking miRNAs in MSC-sEV therapeutics: implications for manufacture, mechanism of action, and development of robust potency CQAs.
   Tan TT, Lim SK. · · 2025 · cited 1× · PMID 41551610 · DOI 10.20517/evcna.2025.55
7. Clinical translational research on stem cell products: prospects and challenges.
   Chen S, Zhang L, Ren Y, Bai X, et al · · 2026 · PMID 42135300 · DOI 10.1038/s41392-026-02582-y
8. The Promise and Challenges of Mesenchymal Stem Cell-Derived Extracellular Vesicles in Periodontal Disease.
   Byun J. · · 2026 · PMID 42075747 · DOI 10.3390/pathogens15040420