Safety and Tolerability Study of a Novel Bioartificial Liver in Liver Failure and Small-for-Size Syndrome

Condition(s) studied

Investigational drug(s) / intervention(s)

Chemically 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

Study 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.

Eligibility

Primary outcome measure(s)

• 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.

Trial sites (1)

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