Hepatocellular CarcinomaMUSE2NCT07786792
Biomarker and Mechanism Study of Systemic Therapy in Unresectable HCC
An observational study of Hepatocellular Carcinoma, run by Shanghai Zhongshan Hospital.
Trial summary
The goal of this observational study is to evaluate the safety and efficacy of systemic therapies and identify predictive molecular biomarkers in patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatocellular Carcinoma
- What is being tested?
- Systemic therapy and Local therapy
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 400 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2026to 1 Jan 2027
- Phase
- Observational
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study population consists of patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) confirmed by histology, cytology, or clinical diagnostic criteria. Eligible patients must have an ECOG performance status of 0 to 2 and well-preserved liver function, defined as Child-Pugh class A or a score of 7. The population includes patients who are scheduled to receive standard systemic therapies, such as targeted therapy combined with immunotherapy or dual immunotherapy, either alone or in combination with local therapies like transcatheter arterial chemoembolization (TACE) or hepatic arterial infusion chemotherapy (HAIC).
Who may be able to join
- Histologically or cytologically confirmed hepatocellular carcinoma (HCC), or meeting the clinical diagnostic criteria of the "Guidelines for Diagnosis and Treatment of Primary Liver Cancer (2024 Edition)" issued by the National Health Commission of China.
- Assessed by the investigator as unsuitable for surgical resection, or having intermediate to advanced stage HCC. Intermediate to advanced HCC is defined as Barcelona Clinic Liver Cancer (BCLC) stage B/C or China Liver Cancer (CNLC) stage IIa/IIb/IIIa/IIIb.
- Child-Pugh liver function class A or a score of 7. Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
- Willing to receive targeted therapy combined with immunotherapy or dual immunotherapy, and signed the informed consent form (applicable to the prospective cohort).
- Adequate bone marrow and organ function, meeting the following laboratory indicators: Absolute neutrophil count (ANC) >= 1.0x10^9/L without the use of granulocyte colony-stimulating factor within the past 14 days; Platelets >= 50x10^9/L without blood transfusion within the past 14 days; Hemoglobin >= 90g/L without blood transfusion or erythropoietin use within the past 14 days; Total bilirubin <= 3 x upper limit of normal (ULN); Albumin >= 28g/L without human serum albumin or plasma infusion within the past 14 days; Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 5 x ULN; Serum creatinine <= 1.5 x ULN; Good coagulation function, defined as an international normalized ratio (INR) of prothrombin time <= 1.5 x ULN.
- Subjects at risk of pregnancy (male or female) must use a contraceptive method with an annual failure rate of less than 1% during the entire treatment period and until 120 days after the last dose of the study drug (or 180 days after the last dose of chemotherapy drugs).
Who cannot join
- Pathological diagnosis of mixed liver cancer, fibrolamellar hepatocellular carcinoma, or other non-hepatocellular malignant tumor components.
- Previous or concurrent other malignant tumors, except for adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix, and papillary thyroid carcinoma.
- History of organ transplantation or hepatic encephalopathy. Known allergy to the selected targeted therapy drugs or immunotherapy drugs or drug excipients, or previous severe allergic reactions to other monoclonal antibodies.
- History of gastrointestinal perforation and/or fistula within the past 6 months, history of intestinal obstruction (including incomplete intestinal obstruction requiring parenteral nutrition), extensive intestinal resection (partial colon resection or extensive small bowel resection complicated by chronic diarrhea), Crohn's disease, ulcerative colitis, or long-term chronic diarrhea.
- Active autoimmune disease requiring systemic treatment (e.g., disease-modifying drugs, corticosteroids, or immunosuppressants) within 2 years prior to the first dose (replacement therapies like thyroxine, insulin, or physiologic corticosteroids for adrenal/pituitary insufficiency are allowed).
- Known history of primary immunodeficiency disease. Uncontrolled hypertension (systolic blood pressure > 140 mmHg or diastolic blood pressure > 90 mmHg after optimal medical treatment), history of hypertensive crisis, or hypertensive encephalopathy.
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- History of esophageal or gastric variceal bleeding events caused by portal hypertension within the past 6 months.
- Diagnosed with severe (G3) varices by digestive endoscopy within 3 months prior to the first dose, or assessed by the investigator as having a high risk of bleeding. Subjects requiring withdrawal of informed consent.
- Other conditions considered unsuitable by the investigator for participation in this clinical trial.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Observational Study and Biomarkers and Molecular Mechanism Study of Systemic Therapy for Intermediate to Advanced Stage or Unresectable Hepatocellular Carcinoma
The goal of this observational study is to evaluate the safety and efficacy of systemic therapies and identify predictive molecular biomarkers in patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC).
The main questions it aims to answer are:What is the objective response rate (ORR) of systemic therapy regimens in this patient population? Which 3-4 specific biomarkers (including tumor or peripheral blood-derived genes, proteins, ctDNA, and immune cell subsets) can reliably predict the efficacy of systemic treatment?
- Conditions
- Hepatocellular Carcinoma
- Being tested
- Systemic therapy, Local therapy
- Keywords
- Hepatocellular Carcinoma, Immunotherapy, Systemic Therapy, Biomarker, ctDNA, Observational Study, Multi-omics
- Medical subject headings
- Carcinoma, Hepatocellular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Systemic therapyDrug
Including regimens such as Atezolizumab + Bevacizumab, Nivolumab + Ipilimumab.
Local therapyProcedure or surgery
Including therapy such as TACE, HAIC
| Study group | Role | What they receive |
|---|---|---|
| Systemic Therapy Cohort | Patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) receiving systemic therapy alone. Receives: Drug: Systemic therapy | |
| TACE/HAIC + Systemic Therapy Cohort | Patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) receiving transcatheter arterial chemoembolization (TACE) or hepatic arterial infusion chemotherapy (HAIC) combined with systemic therapy. Receives: Drug: Systemic therapy; Procedure: Local therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR) Assessed by RECIST v1.1 and HCC mRECIST Criteria
The ORR will be evaluated according to RECIST v1.1 and HCC mRECIST criteria, defined as the sum of complete response (CR) and partial response (PR) in patients with at least one measurable lesion.defined as the sum of complete response (CR) and partial response (PR) in patients with at least one measurable lesion.
Measured: Up to 2 years (assessed every 8 ± 2 weeks)
Levels of Predictive Biomarkers Assessed by Multi-Omics Sequencing and Single Cell RNA Sequencing
Identify 3-4 predictive biomarkers for the efficacy of HCC systemic therapy, including genes, proteins, ctDNA, and immune cell subsets based on tumor tissue or peripheral blood.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study start1 Sep 2026 (estimated)
- Primary completion1 Jan 2027 (estimated)
- Study completion1 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by Shanghai Zhongshan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Zhongshan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hui-Chuan Sun, Shanghai Zhongshan Hospital
- Investigators
- Huichuan Sun, MD, Principal investigator, Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07786792
- Sponsor's protocol ID
- B2026-158R
- Acronym
- MUSE2
- Status last verified
- Aug 2026
- First submitted
- 23 Aug 2026
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study and Biomarkers and Molecular Mechanism Study of Systemic Therapy for Intermediate to Advanced Stage or Unresectable Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT07786792
- Acronym / sponsor ID
- MUSE2 / B2026-158R
- Conditions
- Hepatocellular Carcinoma
- Keywords
- Hepatocellular Carcinoma, Immunotherapy, Systemic Therapy, Biomarker, ctDNA, Observational Study, Multi-omics
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Shanghai Zhongshan HospitalUniversity, hospital or other
- Study start
- 1 Sep 2026 (estimated)
- Primary completion
- 1 Jan 2027 (estimated)
- Study completion
- 1 Jan 2027 (estimated)
- First posted
- 26 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this observational study is to evaluate the safety and efficacy of systemic therapies and identify predictive molecular biomarkers in patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC).
The main questions it aims to answer are:What is the objective response rate (ORR) of systemic therapy regimens in this patient population? Which 3-4 specific biomarkers (including tumor or peripheral blood-derived genes, proteins, ctDNA, and immune cell subsets) can reliably predict the efficacy of systemic treatment?
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Systemic Therapy Cohort | – | Drug: Systemic therapyPatients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) receiving systemic therapy alone. |
| TACE/HAIC + Systemic Therapy Cohort | – | Drug: Systemic therapy, Procedure: Local therapyPatients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) receiving transcatheter arterial chemoembolization (TACE) or hepatic arterial infusion chemotherapy (HAIC) combined with systemic therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population consists of patients with intermediate to advanced stage or unresectable hepatocellular carcinoma (HCC) confirmed by histology, cytology, or clinical diagnostic criteria. Eligible patients must have an ECOG performance status of 0 to 2 and well-preserved liver function, defined as Child-Pugh class A or a score of 7. The population includes patients who are scheduled to receive standard systemic therapies, such as targeted therapy combined with immunotherapy or dual immunotherapy, either alone or in combination with local therapies like transcatheter arterial chemoembolization (TACE) or hepatic arterial infusion chemotherapy (HAIC).
Key inclusion criteria (6 listed)
- Histologically or cytologically confirmed hepatocellular carcinoma (HCC), or meeting the clinical diagnostic criteria of the "Guidelines for Diagnosis and Treatment of Primary Liver Cancer (2024 Edition)" issued by the National Health Commission of China.
- Assessed by the investigator as unsuitable for surgical resection, or having intermediate to advanced stage HCC. Intermediate to advanced HCC is defined as Barcelona Clinic Liver Cancer (BCLC) stage B/C or China Liver Cancer (CNLC) stage IIa/IIb/IIIa/IIIb.
- Child-Pugh liver function class A or a score of 7. Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
- Willing to receive targeted therapy combined with immunotherapy or dual immunotherapy, and signed the informed consent form (applicable to the prospective cohort).
- Adequate bone marrow and organ function, meeting the following laboratory indicators: Absolute neutrophil count (ANC) >= 1.0x10^9/L without the use of granulocyte colony-stimulating factor within the past 14 days; Platelets >= 50x10^9/L without blood transfusion within the past 14 days; Hemoglobin >= 90g/L without blood transfusion or erythropoietin use within the past 14 days; Total bilirubin <= 3 x upper limit of normal (ULN); Albumin >= 28g/L without human serum albumin or plasma infusion within the past 14 days; Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 5 x ULN; Serum creatinine <= 1.5 x ULN; Good coagulation function, defined as an international normalized ratio (INR) of prothrombin time <= 1.5 x ULN.
Key exclusion criteria (9 listed)
- Pathological diagnosis of mixed liver cancer, fibrolamellar hepatocellular carcinoma, or other non-hepatocellular malignant tumor components.
- Previous or concurrent other malignant tumors, except for adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix, and papillary thyroid carcinoma.
- History of organ transplantation or hepatic encephalopathy. Known allergy to the selected targeted therapy drugs or immunotherapy drugs or drug excipients, or previous severe allergic reactions to other monoclonal antibodies.
- History of gastrointestinal perforation and/or fistula within the past 6 months, history of intestinal obstruction (including incomplete intestinal obstruction requiring parenteral nutrition), extensive intestinal resection (partial colon resection or extensive small bowel resection complicated by chronic diarrhea), Crohn's disease, ulcerative colitis, or long-term chronic diarrhea.
- Active autoimmune disease requiring systemic treatment (e.g., disease-modifying drugs, corticosteroids, or immunosuppressants) within 2 years prior to the first dose (replacement therapies like thyroxine, insulin, or physiologic corticosteroids for adrenal/pituitary insufficiency are allowed).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Systemic therapyIncluding regimens such as Atezolizumab + Bevacizumab, Nivolumab + Ipilimumab. | Drug | Systemic Therapy Cohort, TACE/HAIC + Systemic Therapy Cohort |
| Local therapyIncluding therapy such as TACE, HAIC | Procedure or surgery | TACE/HAIC + Systemic Therapy Cohort |
Primary outcome
Objective Response Rate (ORR) Assessed by RECIST v1.1 and HCC mRECIST Criteria
Time frame: Up to 2 years (assessed every 8 ± 2 weeks)
The ORR will be evaluated according to RECIST v1.1 and HCC mRECIST criteria, defined as the sum of complete response (CR) and partial response (PR) in patients with at least one measurable lesion.defined as the sum of complete response (CR) and partial response (PR) in patients with at least one measurable lesion.
Levels of Predictive Biomarkers Assessed by Multi-Omics Sequencing and Single Cell RNA Sequencing
Time frame: Up to 2 years
Identify 3-4 predictive biomarkers for the efficacy of HCC systemic therapy, including genes, proteins, ctDNA, and immune cell subsets based on tumor tissue or peripheral blood.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Huichuan Sun, MD principal investigator
Fudan University
Questions about this trial
What is this clinical trial studying?
An observational study of Hepatocellular Carcinoma, run by Shanghai Zhongshan Hospital.
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 6 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: opening soon, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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.
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?
Contact Hui Li, MD (+86 64041990, li.hui1@zs-hospital.sh.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) Assessed by RECIST v1.1 and HCC mRECIST Criteria (time frame: Up to 2 years (assessed every 8 ± 2 weeks)). The study lists 2 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 2 Sep 2026; AzTrials last refreshed its copy on 7 Nov 2026.