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

NCT07312864 | Recruiting Phase 1

• 1 other identifier: BFH20251124001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Small-for-Size Syndrome; Liver Failure

Outcome Measures

Primary Outcomes (3)

• 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.

  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).

• Survival and Liver Transplantation Rate

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

  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,).

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

  Changes in MELD/PELD score over time will be used to evaluate disease severity and treatment response.

  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)

Secondary Outcomes (21)

• 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).

• 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).

• 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).

Study Arms (1)

CiPS-BAL

EXPERIMENTAL

Chemically induced pluripotent stem cells biological Artificial Liver

Biological: Chemically induced pluripotent stem cells biological Artificial Liver

Interventions

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

CiPS-BAL

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients diagnosed with liver failure (including acute, subacute/acute-on-chronic, and chronic liver failure) or small-for-size syndrome

You may not qualify if:

• 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

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

Study Sites (1)

Beijing Friendship Hospital

Beijing, 101102, China

RECRUITING

MeSH Terms

Conditions

Liver Failure

Condition Hierarchy (Ancestors)

Hepatic Insufficiency; Liver Diseases; Digestive System Diseases

Central Study Contacts

Wan-Ting Zhang

CONTACT

+86 13699189579; 13699189579@163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Department of Critical Liver Diseases

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: December 31, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: April 20, 2026

Record last verified: 2025-11

Locations

CN (1)