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NCT Number: NCT07312864

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

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.

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Key information

Conditions

Liver Failure Digestive System Diseases Hepatic Insufficiency Liver Diseases Small-for-size Syndrome

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Chemically induced pluripotent stem cells biological Artificial Liver

Biological

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

Primary outcomes

  1. Number of participants with treatment-emergent adverse events and serious adverse events

    Time frame: 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.

  2. Survival and Liver Transplantation Rate

    Time frame: 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.

  3. Model for End-Stage Liver Disease (MELD) score/Pediatric End-Stage Liver Disease (PELD) score

    Time frame: 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 outcomes

  1. Glasgow Coma Scale (GCS)

    Time frame: 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.

  2. SOFA Score

    Time frame: 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.

  3. RBC (Red Blood Cell Count)

    Time frame: 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).

  4. Hemoglobin (Hb)

    Time frame: 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).

  5. Blood Ammonia

    Time frame: 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).

  6. Lactate

    Time frame: 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.

  7. WBC (White Blood Cell Count)

    Time frame: 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).

  8. Platelet Count (PLT)

    Time frame: 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).

  9. ALT (Alanine Aminotransferase)

    Time frame: 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).

  10. AST (Aspartate Aminotransferase)

    Time frame: 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).

  11. Total Bilirubin (TBIL)

    Time frame: 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).

  12. Direct Bilirubin (DBIL)

    Time frame: 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).

  13. ALP (Alkaline Phosphatase)

    Time frame: 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).

  14. BUN (Blood Urea Nitrogen)

    Time frame: 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).

  15. Serum Creatinine (Scr)

    Time frame: 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).

  16. Albumin (ALB)

    Time frame: 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).

  17. Glucose (GLU)

    Time frame: 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).

  18. Prothrombin Time (PT)

    Time frame: 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).

  19. Prothrombin Activity (PTA)

    Time frame: 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).

  20. International Normalized Ratio (INR)

    Time frame: 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).

  21. Activated Partial Thromboplastin Time (APTT)

    Time frame: 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).

Study contacts

Contact information is provided by the study sponsor or research team.

Wan-Ting Zhang

CONTACT

[email protected]

+86 13699189579

Sponsors and collaborators

Lead sponsor

Beijing Friendship Hospital

Other

Registry information

Official study title

A Clinical Trial Assessing the Safety, Tolerability, and Exploratory Efficacy of a Novel Bioartificial Liver Therapy in Patients With Liver Failure or Small-for-Size Syndrome

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Dec 31, 2025
Registry last updated
Apr 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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