Liver Failure, Small-for-Size Syndrome
Conditions
Brief summary
The goal of this clinical trial is to evaluate the safety and tolerability of a novel bioartificial liver (CiPS-BAL) in patients with liver failure or small-for-size syndrome. The study will also collect preliminary data on clinical outcomes and laboratory parameters during treatment. The main questions it aims to answer are: Is the novel bioartificial liver system safe and well tolerated in patients with liver failure or small-for-size syndrome? What effects does the treatment have on liver function and other clinical and laboratory indicators? Researchers will treat participants with the CiPS-BAL system, which uses hepatocytes derived from chemically induced pluripotent stem cells (CiPS) within a bioartificial liver device.
Interventions
BIOLOGICALChemically induced pluripotent stem cells biological Artificial Liver
Participants will receive CiPS-BAL therapy, a novel bioartificial liver treatment that combines functional hepatocytes derived from chemically induced pluripotent stem cells (CiPS) with an extracorporeal bioartificial liver device. The CiPS-derived hepatocytes are generated and cultured in vitro under Good Manufacturing Practice (GMP) conditions and subsequently loaded into the bioartificial liver device prior to treatment. Each treatment session utilizes 1 × 10¹⁰ functional hepatocytes. Therapy is administered via central venous access (e.g., femoral, internal jugular, or subclavian vein) for 4-8 hours per session. The planned treatment frequency is one session, with the possibility of additional sessions depending on clinical response and safety evaluation. Standard medical therapy for liver failure or small-for-size syndrome will be provided concomitantly. Participants will be closely monitored for safety, tolerability, and changes in clinical and laboratory parameters throughout
Sponsors
Beijing Friendship Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients diagnosed with liver failure (including acute, subacute/acute-on-chronic, and chronic liver failure) or small-for-size syndrome
Exclusion criteria
* Presence of severe extrahepatic systemic end-stage diseases * Uncontrollable infection or active bleeding * Pregnant or breastfeeding women * History of allergy or known severe hypersensitivity to CiPSC-derived cell products or blood products * Peripheral vascular collapse leading to inability to obtain venous access or collect blood * Unable or unwilling to provide informed consent or unable to comply with study requirements * Unwilling to receive CiPSC-based therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-emergent adverse events and serious adverse events | From completion of CiPS-BAL therapy through Week 4 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4). | The number and proportion of participants experiencing treatment-emergent adverse events (AEs) and serious adverse events (SAEs) following CiPS-BAL therapy, including but not limited to fever, rash, chest tightness, palpitations, acute infusion reactions, immune rejection, infections, and local complications (e.g., hematoma, bleeding), and thrombosis. Adverse events will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. |
| Survival and Liver Transplantation Rate | From completion of CiPS-BAL therapy through Week 4 post-treatment (assessed at 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4,). | Overall survival of the patients and the proportion of participants who undergo liver transplantation during the study period. |
| Model for End-Stage Liver Disease (MELD) score/Pediatric End-Stage Liver Disease (PELD) score | From completion of CiPS-BAL therapy through Week 4 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4) | Changes in MELD/PELD score over time will be used to evaluate disease severity and treatment response. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Glasgow Coma Scale (GCS) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | GCS will be evaluated at each follow-up visit. The outcome will be reported as the change from baseline in GCS score. |
| SOFA Score | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | SOFA score will be measured at each specified time point. The outcome will be reported as the change from baseline in SOFA score. |
| RBC (Red Blood Cell Count) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Hemoglobin (Hb) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Blood Ammonia | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Lactate | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | Lactate levels will be measured from blood samples. The outcome will be reported as the change from baseline at each specified time point. |
| WBC (White Blood Cell Count) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Platelet Count (PLT) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| ALT (Alanine Aminotransferase) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| AST (Aspartate Aminotransferase) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Total Bilirubin (TBIL) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Direct Bilirubin (DBIL) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| ALP (Alkaline Phosphatase) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| BUN (Blood Urea Nitrogen) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Serum Creatinine (Scr) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Albumin (ALB) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Glucose (GLU) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Prothrombin Time (PT) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Prothrombin Activity (PTA) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| International Normalized Ratio (INR) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
| Activated Partial Thromboplastin Time (APTT) | From completion of CiPS-BAL therapy through Week 12 post-treatment (assessed at 1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, and Week 12). | — |
Countries
China
Contacts
CONTACTWan-Ting Zhang
Outcome results
None listed