Hepatocyte-like cell administration via rectus sheath intramuscular injection
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.
Cell quantity: The prespecified therapeutic dose is 1.2×10⁸/kg. To ensure safety, the first patient receives 50% of that dose (0.6×10⁸/kg). After safety confirmation, subsequent patients receive the standard 1.2×10⁸/kg (adjusted based on individual rectus sheath capacity, with actual dose recorded).
Study summary
To evaluate the safety, tolerability, and preliminary efficacy of rectus sheath injection of hepatocyte-like cells in the treatment and prevention of small-for-size syndrome, with the ultimate goal of improving patient survival.
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Incidence of adverse events related to hepatocyte-like cell injection — within 7 days after injection Defined as all adverse events clearly associated with the injection procedure occurring within 7 days after injection, including puncture-site hematoma, secondary infection, etc. The estimated incidence of injection-related adverse events is no more than 25%.
Plasma Ammonia — Blood samples will be collected at before treatment, 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-treatment. Ammonia levels in μmol/L will be measured from blood samples. The outcome will be reported as the change from baseline in plasma ammonia levels at each specified time point.
Serum Direct Bilirubin — Blood samples will be collected at before treatment, 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-treatment. Direct bilirubin levels in mg/dL (or μmol/L) will be measured from blood samples. The outcome will be reported as the change from baseline in direct bilirubin levels at each specified time point.
Serum Total Bilirubin — Blood samples will be collected at before treatment, 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-treatment. Total bilirubin levels in mg/dL (or μmol/L) will be measured from blood samples. The outcome will be reported as the change from baseline in total bilirubin levels at each specified time point.
Serum Aspartate Aminotransferase (AST) — Blood samples will be collected at before treatment, 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-treatment. AST levels in U/L will be measured from blood samples. The outcome will be reported as the change from baseline in AST levels at each specified time point.
Serum Alanine Aminotransferase (ALT) — Blood samples will be collected at before treatment, 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-treatment. ALT levels in U/L will be measured from blood samples. The outcome will be reported as the change from baseline in ALT levels at each specified time point.
Incidence of short-term adverse events related to the hepatocyte-like cell product — within 1 month after injection Defined as adverse events clearly associated with the hepatocyte-like cell product occurring within 1 month after injection, including fever, rash, hypotension, allergic reactions, etc. The estimated incidence of short-term adverse events related to the hepatocyte-like cell product is no more than 25%.
Incidence, severity, and prognosis of small-for-size syndrome — Within 3 months after injection The incidence, severity grade, and patient prognosis of small-for-size syndrome will be recorded, using patients who did not receive hepatocyte-like cell injection during the same period as historical controls.
Coagulation Function — Blood samples will be collected before injection, at 6 hours, 12 hours, and on days 1, 3, 7, 14, as well as at months 1, 2, and 3 post-injection. (including Prothrombin Time \[PT\], Activated Partial Thromboplastin Time \[APTT\], International Normalized Ratio \[INR\], and Prothrombin Time Activity \[PTA\]). PT and APTT will be measured in seconds, INR will be reported as a ratio, and PTA will be measured as a percentage (%). The outcome will be reported as the change from baseline in each coagulation parameter at each specified time point.
Trial sites (1)
Facility
City
Region
Status
Beijing Friendship Hospital, Capital Medical University
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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