Tauroursodeoxycholic Acid Combined With PD-1/PD-L1 Immunotherapy in Advanced Hepatocellular Carcinoma: A Prospective Study

NCT ID: NCT07064668

Last Updated: 2025-07-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

300 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-07-30

Study Completion Date

2027-12-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is a clinical trial that aims to evaluate whether adding a bile acid called tauroursodeoxycholic acid (TUDCA) can improve the effects of immunotherapy in patients with advanced liver cancer (hepatocellular carcinoma).

Immunotherapy has shown promise in treating this type of cancer, but not all patients respond well. TUDCA is known to help protect liver cells and may improve the liver's immune environment, potentially making immunotherapy more effective.

In this study, 300 patients with advanced liver cancer will be randomly assigned to receive either immunotherapy alone or immunotherapy combined with TUDCA.

Researchers will look at how well the cancer responds, whether the treatment helps more patients become eligible for surgery, and how safe the combination is.

The goal is to find a more effective and better tolerated treatment for patients with liver cancer.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This prospective, randomized, controlled clinical trial investigates the efficacy and safety of combining tauroursodeoxycholic acid (TUDCA) with immune checkpoint inhibitors (ICIs) in patients with advanced hepatocellular carcinoma (HCC).

Immunotherapy using PD-1 or PD-L1 inhibitors has become a cornerstone in treating advanced HCC. However, a significant proportion of patients exhibit limited response or develop resistance. One contributing factor may be the immunosuppressive liver tumor microenvironment, which impairs T cell activation and infiltration. TUDCA is a hydrophilic bile acid that exhibits cytoprotective and anti-inflammatory effects, and may enhance the immune response by improving liver immune homeostasis.

In this study, 300 patients with Barcelona Clinic Liver Cancer (BCLC) stage C HCC will be enrolled and randomly assigned to receive either PD-1/PD-L1 inhibitor monotherapy or combination therapy with TUDCA. The treatment cycle will last for 6 months, with a follow-up period of at least 6 months after treatment completion.

The primary outcome is objective response rate (ORR) as measured by RECIST 1.1 and mRECIST criteria. Secondary endpoints include progression-free survival (PFS), overall survival (OS), conversion to surgical eligibility, and biomarker dynamics.

Safety and tolerability will be closely monitored throughout the study. Dose-limiting toxicities, adverse events, and liver function parameters will be assessed regularly. The trial will also explore immune and inflammatory biomarkers associated with response to treatment.

This study aims to determine whether the addition of TUDCA can enhance the efficacy of ICIs in HCC and provide a novel treatment strategy for patients with advanced disease.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hepatocellular Carcinoma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a randomized, parallel assignment, controlled interventional study. Eligible participants with advanced hepatocellular carcinoma (BCLC stage C) will be randomly assigned in a 1:1 ratio to receive either standard immune checkpoint inhibitor (ICI) monotherapy or ICI therapy in combination with tauroursodeoxycholic acid (TUDCA). The study will assess efficacy and safety over a 6-month treatment period with follow-up extending to 12 months post-treatment. No crossover between arms is planned.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

TUDCA + ICI

Participants will receive a combination of tauroursodeoxycholic acid (TUDCA) and an immune checkpoint inhibitor.

Group Type EXPERIMENTAL

TUDCA (Tauroursodeoxycholic Acid) Supplementation

Intervention Type DRUG

TUDCA administered orally according to protocol-defined dosage.

Immune checkpoint inhibitor (ICI)

Intervention Type DRUG

Administered intravenously according to standard practice or protocol.

ICI Monotherapy

Participants will receive standard immune checkpoint inhibitor monotherapy.

Group Type ACTIVE_COMPARATOR

Immune checkpoint inhibitor (ICI)

Intervention Type DRUG

Administered intravenously according to standard practice or protocol.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

TUDCA (Tauroursodeoxycholic Acid) Supplementation

TUDCA administered orally according to protocol-defined dosage.

Intervention Type DRUG

Immune checkpoint inhibitor (ICI)

Administered intravenously according to standard practice or protocol.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Histologically or cytologically confirmed diagnosis of primary hepatocellular carcinoma (HCC).

Unresectable advanced HCC classified as Barcelona Clinic Liver Cancer (BCLC) stage C.

Age ≥ 18 years.

No prior systemic anti-tumor therapy for HCC before first dose of study treatment.

At least one measurable lesion according to RECIST v1.1, or a measurable lesion with clear progression after local treatment based on RECIST v1.1.

Adequate organ and bone marrow function within 7 days prior to enrollment, with no blood products, growth factors, albumin, or other intravenous/subcutaneous corrective treatment within 14 days prior to laboratory assessment:

Hematology:

Absolute neutrophil count (ANC) ≥ 1.5 × 10⁹/L Platelet count (PLT) ≥ 75 × 10⁹/L Hemoglobin (Hb) ≥ 9.0 g/dL

Liver function:

Total bilirubin (TBil) ≤ 2 × ULN ALT and AST ≤ 5 × ULN Serum albumin ≥ 28 g/L

Alkaline phosphatase (ALP) ≤ 5 × ULN

Renal function:

Serum creatinine (Cr) ≤ 1.5 × ULN or creatinine clearance (CCr) ≥ 50 mL/min (calculated by Cockcroft-Gault formula) Urinalysis shows urine protein \< 2+; if urine protein ≥ 2+, 24-hour urine collection must show protein \< 1g/24h

Coagulation:

International Normalized Ratio (INR) ≤ 2.3 or prothrombin time (PT) prolongation ≤ 6 seconds

Estimated life expectancy of ≥ 12 weeks.

Exclusion Criteria

History of hepatic encephalopathy or liver transplantation.

Acute cholecystitis, acute cholangitis, patients with frequent biliary colic attacks, complete biliary obstruction, or gallbladder dysfunction (inability to contract and empty).

Patients with Child-Pugh class C or decompensated cirrhosis, including those with a history of severe hepatic encephalopathy or refractory ascites due to liver disease.

Patients with a previous diagnosis of biliary atresia without adequate biliary drainage (e.g., failed biliary-enteric anastomosis).

Pregnant or breastfeeding women.
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Tongji Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Zhang ZhanGuo

Prof.

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Tongji Medical college of HUST

Wuhan, Hubei, China

Site Status

Countries

Review the countries where the study has at least one active or historical site.

China

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Zhanguo Zhang, doctor

Role: CONTACT

+8613517726223

Shangwu Ning, master

Role: CONTACT

+8613517726223

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Zhanguo Zhang, doctor

Role: primary

+8613517726223

References

Explore related publications, articles, or registry entries linked to this study.

Lan X, Ma J, Huang Z, Xu Y, Hu Y. Akkermansia muciniphila might improve anti-PD-1 therapy against HCC by changing host bile acid metabolism. J Gene Med. 2024 Jan;26(1):e3639. doi: 10.1002/jgm.3639. Epub 2023 Dec 7.

Reference Type BACKGROUND
PMID: 38058259 (View on PubMed)

Kusaczuk M. Tauroursodeoxycholate-Bile Acid with Chaperoning Activity: Molecular and Cellular Effects and Therapeutic Perspectives. Cells. 2019 Nov 20;8(12):1471. doi: 10.3390/cells8121471.

Reference Type BACKGROUND
PMID: 31757001 (View on PubMed)

Latif MU, Schmidt GE, Mercan S, Rahman R, Gibhardt CS, Stejerean-Todoran I, Reutlinger K, Hessmann E, Singh SK, Moeed A, Rehman A, Butt UJ, Bohnenberger H, Stroebel P, Bremer SC, Neesse A, Bogeski I, Ellenrieder V. NFATc1 signaling drives chronic ER stress responses to promote NAFLD progression. Gut. 2022 Dec;71(12):2561-2573. doi: 10.1136/gutjnl-2021-325013. Epub 2022 Apr 1.

Reference Type BACKGROUND
PMID: 35365570 (View on PubMed)

Oura K, Morishita A, Tani J, Masaki T. Tumor Immune Microenvironment and Immunosuppressive Therapy in Hepatocellular Carcinoma: A Review. Int J Mol Sci. 2021 May 28;22(11):5801. doi: 10.3390/ijms22115801.

Reference Type BACKGROUND
PMID: 34071550 (View on PubMed)

Zheng J, Wang S, Xia L, Sun Z, Chan KM, Bernards R, Qin W, Chen J, Xia Q, Jin H. Hepatocellular carcinoma: signaling pathways and therapeutic advances. Signal Transduct Target Ther. 2025 Feb 7;10(1):35. doi: 10.1038/s41392-024-02075-w.

Reference Type BACKGROUND
PMID: 39915447 (View on PubMed)

Cunningham M, Gupta R, Butler M. Checkpoint inhibitor hepatotoxicity: pathogenesis and management. Hepatology. 2024 Jan 1;79(1):198-212. doi: 10.1097/HEP.0000000000000045. Epub 2023 Jan 13.

Reference Type BACKGROUND
PMID: 36633259 (View on PubMed)

Fleishman JS, Kumar S. Bile acid metabolism and signaling in health and disease: molecular mechanisms and therapeutic targets. Signal Transduct Target Ther. 2024 Apr 26;9(1):97. doi: 10.1038/s41392-024-01811-6.

Reference Type BACKGROUND
PMID: 38664391 (View on PubMed)

Feng L, Zhang W, Shen Q, Miao C, Chen L, Li Y, Gu X, Fan M, Ma Y, Wang H, Liu X, Zhang X. Bile acid metabolism dysregulation associates with cancer cachexia: roles of liver and gut microbiome. J Cachexia Sarcopenia Muscle. 2021 Dec;12(6):1553-1569. doi: 10.1002/jcsm.12798. Epub 2021 Sep 28.

Reference Type BACKGROUND
PMID: 34585527 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

TJ-IRB202505056

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Phase 2 Study of WGI-0301 for Advanced HCC
NCT06309485 NOT_YET_RECRUITING PHASE2