Safety and Tolerability of Hepatocyte-like Cell Therapy for Liver Failure and Small-for-Size Syndrome

NCT ID: NCT07276568

Last Updated: 2025-12-11

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-01
• Study Completion Date: 2029-12-31

Brief Summary

To evaluate the safety, tolerability, and preliminary efficacy of intramuscular injection of hepatocyte-like cells into the rectus sheath in patients with liver failure (including acute liver failure, acute-on-chronic liver failure, and chronic liver failure) and small-for-size syndrome, with the ultimate goal of improving survival rates.

Conditions

Acute Liver Failure; Acute on Chronic Liver Failure (ACLF); Chronic Liver Failure; Small-for-size Syndrome

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Acute Liver Failure

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Group Type: EXPERIMENTAL

Hepatocyte-like cell administration via rectus sheath intramuscular injection

Intervention Type: DRUG

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Acute-on-Chronic Liver Failure

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Group Type: EXPERIMENTAL

Hepatocyte-like cell administration via rectus sheath intramuscular injection

Intervention Type: DRUG

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Chronic liver failure

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Group Type: EXPERIMENTAL

Hepatocyte-like cell administration via rectus sheath intramuscular injection

Intervention Type: DRUG

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Small-for-Size Syndrome

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Group Type: EXPERIMENTAL

Hepatocyte-like cell administration via rectus sheath intramuscular injection

Intervention Type: DRUG

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Interventions

Hepatocyte-like cell administration via rectus sheath intramuscular injection

On the basis of standard medical therapy, patients will receive an intramuscular injection of CiPS-derived hepatocyte cells into the rectus sheath.

The method, dose, timing, and treatment course of rectus sheath injection of CiPS-derived hepatocyte cells (adjusted by body weight for adults/adolescents) are set as follows:

① Cell quantity: A dose-escalation design (ranging from 1.6 × 10⁷/kg to 1.7 × 10⁸/kg) will be adopted. Treatment will initiate at the lower dose (1.6 × 10⁷/kg). Upon successful safety review, the dose will be gradually escalated up to a maximum of 1.7 × 10⁸/kg.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Patients must be diagnosed with liver failure or small-for-size syndrome based on clinical presentation.

（Acute Liver Failure (ALF) An acute onset of liver failure characterized by hepatic encephalopathy of grade II or higher within 4 weeks, in a patient without pre-existing liver disease.

Acute-on-Chronic Liver Failure (ACLF) A complex clinical syndrome characterized by acute deterioration of liver function, triggered by precipitating events, in patients with underlying chronic liver disease (with or without cirrhosis). It manifests as single or multiple organ failure(s) and is associated with high short-term mortality (28-day mortality rate ≥ 15%).

Chronic Liver Failure (CLF) A state of chronic hepatic decompensation occurring progressively in patients with cirrhosis. It is primarily characterized by recurrent ascites and/or hepatic encephalopathy resulting from progressively declining liver function.

Small-for-Size Syndrome (SFSS) Diagnosis is established according to the International Liver Transplantation Society (ILTS) 2023 guidelines on SFSS.） ② Patients must agree to undergo intramuscular injection of cells into the rectus sheath.

Exclusion Criteria

-

Subjects meeting any of the following criteria will be excluded from the study:

1. Presence of severe, life-threatening extrahepatic systemic diseases.

2. Uncontrolled active infection or hemorrhage.

3. Pregnancy or lactation in female patients.

4. History of severe allergic reactions or known hypersensitivity to CiPS-derived cell products or blood products.

5. Inability to undergo phlebotomy due to peripheral vascular collapse.

6. Inability or unwillingness to provide informed consent or comply with the study procedures.

7. Unwillingness to receive CiPS-based therapy.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Zhi-Jun Zhu

OTHER

Sponsor Role: lead

Responsible Party

Zhi-Jun Zhu

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Central Contacts

Ruzhou Cai

Role: CONTACT

18722563616@126.com

+8618722563616

Other Identifiers

BFH20251030001

Identifier Type: -

Identifier Source: org_study_id