Safety, Tolerability, and Preliminary Efficacy of Hepatocyte-like Cell Injection for the Prevention and Treatment of Small-for-Size Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Hepatocyte-like cell administration via rectus sheath intramuscular injection.
- Who it may be relevant to
- Registry conditions: Small-for-size Syndrome. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Clinical Study on the Safety, Tolerability, and Preliminary Efficacy of Hepatocyte-like Cell Injection for the Prevention and Treatment of Small-for-Size Syndrome
Overview
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.
Interventions
- Drug 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).
Primary outcome measures
- Incidence of adverse events related to hepatocyte-like cell injection [Time frame: within 7 days after injection]
- Plasma Ammonia [Time frame: 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.]
- Serum Direct Bilirubin [Time frame: 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.]
- Serum Total Bilirubin [Time frame: 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.]
- Serum Aspartate Aminotransferase (AST) [Time frame: 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.]
- Serum Alanine Aminotransferase (ALT) [Time frame: 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.]
- Incidence of short-term adverse events related to the hepatocyte-like cell product [Time frame: within 1 month after injection]
- Incidence, severity, and prognosis of small-for-size syndrome [Time frame: Within 3 months after injection]
- Coagulation Function [Time frame: 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.]
Secondary outcome measures (10)
- Heart Rate [Time frame: Measured at before treatment, and at 1, 3, 6, 12 hours post-treatment, and on days 1, 3, 7, 14, as well as at months 1, 2 and 3 post-treatment.]
- Respiratory Rate [Time frame: Measured at before treatment, and at 1, 3, 6, 12 hours post-treatment, and on days 1, 3, 7, 14, as well as at months 1, 2 and 3 post-treatment.]
- Blood Pressure [Time frame: Measured at before treatment, and at 1, 3, 6, 12 hours post-treatment, and on days 1, 3, 7, 14, as well as at months 1, 2 and 3 post-treatment.]
- Graft Morphological Stability on T2-weighted Fat-Suppressed (T2W-FS) MRI [Time frame: MRI assessments will be performed at baseline (pre-treatment), and at Day 7, 14, Month 1, 2, and 3 post-treatment.]
- Graft Signal Characteristics on T2-weighted Fat-Suppressed (T2W-FS) MRI [Time frame: MRI assessments will be performed at baseline (pre-treatment), and at Day 7, 14, Month 1, 2, and 3 post-treatment.]
- Absence of Abnormal Fat Deposition on T1-weighted Fat-Suppressed (T1W-FS) MRI [Time frame: MRI assessments will be performed at baseline (post-transplantation), and at Day 28, Month 3, 6, and 12 post-transplantation.]
- Rectus Sheath Ultrasound Examination [Time frame: Before injection, 12 hours post-injection, day 3, day 7 , day 14, and months 1, 2, and 3.]
- Change from baseline in serum levels of tumor markers measured in ng/mL [Time frame: Blood samples will be collected before injection, and on days 14, as well as at months 1, 2, and 3 post-injection.]
- Change from baseline in serum levels of tumor markers measured in μg/L [Time frame: Blood samples collected before injection, and on day 14, as well as at months 1, 2, and 3 post-injection.]
- Incidence of medium-to-long-term adverse events related to the hepatocyte-like cell product [Time frame: 1to 3 month after injection]
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:
- Presence of severe, life-threatening extrahepatic systemic diseases.
- Uncontrolled active infection or hemorrhage.
- Pregnancy or lactation in female patients.
- History of severe allergic reactions or known hypersensitivity to CiPS-derived cell products or blood products.
- Inability to undergo phlebotomy due to peripheral vascular collapse.
- Inability or unwillingness to provide informed consent or comply with the study procedures.
- Unwillingness to receive CiPS-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
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Beijing Friendship Hospital, Capital Medical University — Beijing
Identifiers
NCT: NCT07276568 · BFH20251030001