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

Official 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

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.

Interventions

• Biological 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. Therap

Primary outcome measures

• 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).]
• 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,).]
• 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)]

Secondary outcome measures (12)

• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]
• 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).]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

• Beijing Friendship Hospital — Beijing

Identifiers

NCT: NCT07312864 · BFH20251124001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗