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Small-for-size SyndromeEarly Phase 1NCT07276568

Safety, Tolerability, and Preliminary Efficacy of Hepatocyte-like Cell Injection for the Prevention and Treatment of Small-for-Size Syndrome

A Early Phase 1 study testing Hepatocyte-like cell administration via rectus sheath intramuscular injection in people with Small-for-size Syndrome, run by Zhi-Jun Zhu at 1 site in China.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Small-for-size Syndrome
What is being tested?
Hepatocyte-like cell administration via rectus sheath intramuscular injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteChina
Study dates
17 Dec 2025to 1 Dec 2029
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • -
  • 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.
Show all 9Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site
  • Beijing Friendship Hospital, Capital Medical University

    Beijing, Province, 100050

Other trials in China

About this study

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

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.

Being tested
Hepatocyte-like cell administration via rectus sheath intramuscular injection

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Hepatocyte-like cell administration via rectus sheath intramuscular injectionDrug

    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 groups in NCT07276568
Study groupRoleWhat they receive
Small-for-Size SyndromeExperimentalOn 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).
Receives: Drug: Hepatocyte-like cell administration via rectus sheath intramuscular injection
High risk of small-for-size syndromeExperimentalOn 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).
Receives: Drug: Hepatocyte-like cell administration via rectus sheath intramuscular injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events related to hepatocyte-like cell 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%.

    Measured: within 7 days after injection

  2. Plasma Ammonia

    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.

    Measured: 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.

  3. Serum Direct Bilirubin

    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.

    Measured: 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.

  4. Serum Total Bilirubin

    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.

    Measured: 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.

  5. Serum Aspartate Aminotransferase (AST)

    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.

    Measured: 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.

  6. Serum Alanine Aminotransferase (ALT)

    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.

    Measured: 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.

  7. Incidence of short-term adverse events related to the hepatocyte-like cell product

    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%.

    Measured: within 1 month after injection

  8. Incidence, severity, and prognosis of small-for-size syndrome

    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.

    Measured: Within 3 months after injection

  9. Coagulation Function

    (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.

    Measured: 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 outcomes (10)
  1. Heart Rate

    Heart rate will be measured in beats per minute (bpm) in a supine or sitting position after the participant has rested for at least 5 minutes. The outcome will be reported as the change from baseline in heart rate at each specified time point.

    Measured: 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.

  2. Respiratory Rate

    Respiratory rate will be measured in breaths per minute (brpm). The outcome will be reported as the change from baseline in respiratory rate at each specified time point.

    Measured: 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.

  3. Blood Pressure

    Blood pressure will be measured in millimeters of mercury (mmHg) in a supine or sitting position after the participant has rested for at least 5 minutes.

    Measured: 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.

  4. Graft Morphological Stability on T2-weighted Fat-Suppressed (T2W-FS) MRI

    The graft site within the left rectus abdominis muscle will be assessed by T2W-FS MRI for morphological stability. The outcome is based on the presence or absence of significant changes in location, shape, and size compared to the baseline (post-engraftment) scan. The absence of mass effect, displacement, or deformation of the surrounding muscle will be documented. Stability in these parameters over time will be considered a favorable outcome, while significant deviation will be reported as an adverse event.

    Measured: MRI assessments will be performed at baseline (pre-treatment), and at Day 7, 14, Month 1, 2, and 3 post-treatment.

  5. Graft Signal Characteristics on T2-weighted Fat-Suppressed (T2W-FS) MRI

    The graft site will be assessed by T2W-FS MRI for signal intensity characteristics. The outcome is based on the signal intensity and pattern (e.g., homogeneous, stippled, nodular) relative to the surrounding normal muscle tissue. A stable or resolving signal pattern without the development of focal, nodular, or mass-like high signal intensity will be considered a favorable outcome. The development of new or evolving high-signal areas suggestive of abnormal growth will be reported.

    Measured: MRI assessments will be performed at baseline (pre-treatment), and at Day 7, 14, Month 1, 2, and 3 post-treatment.

  6. Absence of Abnormal Fat Deposition on T1-weighted Fat-Suppressed (T1W-FS) MRI

    The graft site will be assessed by T1W-FS MRI for the absence of abnormal fat deposition, a key indicator for teratoma formation. The outcome is a binary assessment (Yes/No) of whether the signal intensity of the graft region is isointense to surrounding normal muscle tissue.

    Measured: MRI assessments will be performed at baseline (post-transplantation), and at Day 28, Month 3, 6, and 12 post-transplantation.

  7. Rectus Sheath Ultrasound Examination

    With the subject in a supine position and the abdomen fully exposed, an ultrasound probe is placed along the orientation of the rectus abdominis muscle, performing longitudinal and transverse scans. The thickness of the rectus sheath (vertical distance from the inner edge of the anterior sheath to the anterior edge of the posterior sheath) is measured. After injection of hepatocyte-like cells, the same measurements are repeated at each follow-up time point. Meanwhile, special attention is paid to the presence of any abnormal echoic areas within the rectus sheath (e.g., hematoma, effusion, abnormal cell aggregation, or space-occupying lesions), and their location, size, margins, and internal echo characteristics are recorded. Color Doppler mode is used to assess whether abnormal blood flow signals are present in the injection area.

    Measured: Before injection, 12 hours post-injection, day 3, day 7 , day 14, and months 1, 2, and 3.

  8. Change from baseline in serum levels of tumor markers measured in ng/mL

    Including Alpha-Fetoprotein (AFP), Carcinoembryonic Antigen (CEA), Cancer Antigen 125 (CA125), Carbohydrate Antigen 19-9 (CA19-9), Total Prostate-Specific Antigen (tPSA), Free Prostate-Specific Antigen (fPSA), Complexed Prostate-Specific Antigen (c-PSA), Carbohydrate Antigen CA242, Carbohydrate Antigen CA724, Carbohydrate Antigen CA50, and Cancer Antigen 15-3 (CA153). For markers specific to males (tPSA, fPSA, c-PSA) or females (CA153), only applicable subjects will be tested.

    Unit of Measure: ng/mL

    Measured: Blood samples will be collected before injection, and on days 14, as well as at months 1, 2, and 3 post-injection.

  9. Change from baseline in serum levels of tumor markers measured in μg/L

    Including Neuron-Specific Enolase (NSE), Cytokeratin 19 Fragment (CYFRA21-1), Squamous Cell Carcinoma Antigen (SCC), and Human Epididymis Protein 4 (HE4). For HE4 (female-specific), only applicable subjects will be tested.

    Unit of Measure: μg/L

    Measured: Blood samples collected before injection, and on day 14, as well as at months 1, 2, and 3 post-injection.

  10. Incidence of medium-to-long-term adverse events related to the hepatocyte-like cell product

    Defined as adverse events clearly associated with the hepatocyte-like cell product occurring between 1 month and 3 months after injection, including non-targeted cell migration, abnormal proliferation, etc. The estimated incidence of medium-to-long-term adverse events related to the hepatocyte-like cell product is no more than 15%.

    Measured: 1to 3 month after injection

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Dec 2025
  2. Study start17 Dec 2025
  3. Primary completion1 Dec 2028 (estimated)
  4. Study completion1 Dec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07276568
Sponsor's protocol ID
BFH20251030001
Status last verified
Nov 2025
First submitted
20 Nov 2025
Last updated
15 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Hepatocyte-like cell administration via rectus sheath intramuscular injection in people with Small-for-size Syndrome, run by Zhi-Jun Zhu at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events related to hepatocyte-like cell injection (time frame: within 7 days after injection). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.

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