Safety and Tolerability Study of a Novel Bioartificial Liver in Liver Failure and Small-for-Size Syndrome
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorBeijing Friendship Hospital
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
Patients diagnosed with liver failure (including acute, subacute/acute-on-chronic, and chronic liver failure) or small-for-size syndrome
Disqualifiers
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
Trial design
Design model
Single group
Treatments tested in this trial
Chemically induced pluripotent stem cells biological Artificial Liver
Biological/VaccineParticipants 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
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: CiPS-BALExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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 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).2
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
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,).3
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.
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 outcomes
1
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.
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).2
SOFA Score
SOFA score will be measured at each specified time point. The outcome will be reported as the change from baseline in 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).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).Sponsors and contacts
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Source ClinicalTrials.gov