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

Официальное название

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

Обзор

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.

Вмешательства

• Биопрепарат 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

Первичные конечные точки

• Number of participants with treatment-emergent adverse events and serious adverse events [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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)]

Вторичные конечные точки (12)

• Glasgow Coma Scale (GCS) [Срок оценки: 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 [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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) [Срок оценки: 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).]

Критерии участия

Критерии включения

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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Beijing Friendship Hospital — Пекин

Идентификаторы

NCT: NCT07312864 · BFH20251124001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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