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

Phase 2RecruitingNCT07312864

Beijing Friendship Hospital12 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Liver Failure Small-for-Size Syndrome

Interventions

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 cultured and induced in vitro under GMP conditions, then loaded into the BAL device for treatment. Therapy is administered via central venous access (e.g., femoral, internal jugular, or subclavian vein) for 4-8 hours per session. Each patient receives approximately 1.67 × 10\^8 functional hepatocytes per kg of body weight, with treatment frequency and duration adjusted according to clinical response. Standard medical care for liver failure or small-for-size syndrome is provided alongside CiPS-BAL therapy. Patients are closely monitored for safety, tolerability, and changes in clinical and laboratory parameters.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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

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.

Time frame: From initiation of CiPS-BAL therapy through 12 months post-treatment (assessed at Day 7, Week 4, Week 12, Month 6, and Month 12)

Survival and Liver Transplantation Rate

Overall survival of the patients and the proportion of participants who undergo liver transplantation during the study period.

Time frame: 7 days, 4 weeks, 12 weeks, 6 months, and 12 months after CiPS-BAL therapy.

Secondary Outcomes

Change from baseline in Model for End-Stage Liver Disease (MELD) score

The MELD score, a validated composite score based on serum bilirubin, international normalized ratio (INR), and serum creatinine, will be assessed at each follow-up visit. The outcome will be reported as the change from baseline in MELD score at each specified time point.

Time frame: Baseline; 0.5-1 hour, 12 hours, 24 hours, Day 2, Day 3, Day 5, Day 7, Week 2, Week 4, Week 6, Week 12, Month 6, and Month 12 after CiPS-BAL therapy

Glasgow Coma Scale (GCS)

GCS will be evaluated at each follow-up visit. The outcome will be reported as the change from baseline in GCS score.