A Phase II Trial of Organoid Drug Sensitivity Testing to Guide Therapy in Unresectable Biliary Tract Cancers

NCT07351591 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: EHBHKY2025-H033-P001
• Study Type: interventional
• Target: 88
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a prospective, non-randomized, open-label, single-center phase II clinical trial. It aims to evaluate the efficacy and feasibility of using patient-derived tumor organoid drug sensitivity testing (ODST) to guide personalized systemic therapy for patients with unresectable biliary tract cancers (BTC). A total of 88 eligible patients will be enrolled and grouped based on patient preference into either the ODST-guided group or the control group (standard therapy). Tumor tissues obtained via biopsy will be used to establish organoid cultures. Drug sensitivity testing will be performed on a panel of approved regimens (including GC, GEMOX, Durvalumab+GC, Pembrolizumab+GC, and Toripalimab+Lenvatinib+GEMOX) to identify the most effective treatment. Patients for whom organoid testing fails or results are unavailable within one month will receive standard therapy. The primary endpoints are Objective Response Rate (ORR) and Progression-Free Survival (PFS). Secondary endpoints include Overall Survival (OS) and safety profiles. The study seeks to provide a novel, personalized treatment strategy to improve outcomes for patients with advanced BTC.

Conditions

Advanced Biliary Tract Cancer(BTC)

Keywords

organoid; drug-sensitive test

Outcome Measures

Primary Outcomes (2)

• Objective Response Rate(ORR)

  ORR was assessed by investigators per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1).

  Through out the study (up to 3 years)

• Progression-free Survival(PFS)

  In treated subjects, the time from first recording to tumor progression (based on RECIST 1.1 and mRECIST) or death from any cause.

  up to 3 years

Secondary Outcomes (2)

• Overall Survival(OS)

  Up to 3 years

• Adverse event

  From the date of each patient's enrollment until 30 days (±7 days) after the last dose of study drug or until the initiation of new anti-tumor therapy, whichever occurs first.

Study Arms (1)

Arm 1

EXPERIMENTAL

Physician's Choice Group：According to empirical and clinical practice

Drug: Organoid Drug Sensitivity Testing-Guided Therapy

Interventions

Organoid Drug Sensitivity Testing-Guided Therapy DRUG

Fresh tumor tissue from resection is used to establish and culture patient-derived organoids. Successful organoids undergo drug sensitivity testing against a predefined panel of drugs (Gemcitabine + Cisplatin,Gemcitabine + Oxaliplatin,Durvalumab + Gemcitabine + Cisplatin,Pembrolizumab + Gemcitabine + Cisplatin,Toripalimab + Lenvatinib + Gemcitabine + Oxaliplatin). The most effective drug(s), based on IC50 and AUC values, are recommended for treatment

Arm 1

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged between 18 and 70 years (inclusive).
• Diagnosis of biliary tract malignancy confirmed according to the NCCN Clinical Practice Guidelines.
• The subject or their legal guardian understands and voluntarily signs the Informed Consent Form, and is willing and able to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures as required by the protocol.
• The subject is not a candidate for curative resection, transplantation, or local ablation therapy. This includes patients with recurrent disease after prior radical therapy who are not eligible for further curative resection or ablation.
• Life expectancy of at least 12 weeks.
• No radiotherapy within 12 weeks prior to the first dose of the study drug.
• Liver function classified as Child-Pugh Class A or Class B with a score of 7.
• Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1.
• Adequate organ and bone marrow function, defined by the following laboratory values within 7 days prior to randomization (without receiving any blood transfusions, hematopoietic growth factors, albumin, or other corrective drugs within 14 days prior to the laboratory tests):
• Hematological:
• Absolute Neutrophil Count (ANC) ≥ 1.5 × 10⁹/L
• Platelet count (PLT) ≥ 75 × 10⁹/L
• Hemoglobin (HGB) ≥ 9.0 g/dL 9.2. Hepatic:
• Total Bilirubin (TBIL) ≤ 3 × Upper Limit of Normal (ULN)
• Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), and Alkaline Phosphatase (ALP) ≤ 5 × ULN

You may not qualify if:

• Uncorrectable coagulopathy or individuals with a significant bleeding tendency.
• Evidence of any concurrent malignant disease.
• Diagnosis of another malignancy within 3 years prior to the first dose, except for radically treated cutaneous basal cell carcinoma, cutaneous squamous cell carcinoma, and/or carcinoma in situ that has undergone curative resection.
• Patients requiring long-term anticoagulant or antiplatelet therapy that cannot be discontinued.
• Presence of hepatic encephalopathy or refractory pleural effusion/ascites requiring therapeutic intervention.
• Other anti-tumor or systemic therapies within 2 weeks prior to enrollment:
• Treatment with Chinese herbal medicine with demonstrated anti-tumor properties.
• Treatment with Chinese herbal medicine with anti-tumor indications or drugs with immunomodulatory effects (including thymosin, interferon, interleukin), except for localized use to control pleural effusion.
• History of systemic treatment for active autoimmune disease or ongoing immunosuppressive therapy:
• Active autoimmune disease requiring systemic treatment (e.g., disease-modifying drugs, corticosteroids, or immunosuppressants) within 2 years prior to the first dose. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement for adrenal or pituitary insufficiency) is not considered systemic treatment.
• Systemic corticosteroid therapy (excluding topical, intranasal, inhaled, or other local routes) or any other form of immunosuppressive therapy within 7 days prior to the first study dose. The use of physiologic doses of corticosteroids (≤10 mg/day prednisone or equivalent) is permitted.
• Severe hepatic or renal insufficiency.
• Presence of any severe or uncontrolled systemic disease, including but not limited to:
• Clinically significant, poorly controlled resting ECG abnormalities (e.g., complete left bundle branch block, second-degree or higher atrioventricular block, ventricular arrhythmia, or atrial fibrillation).
• Unstable angina, congestive heart failure (New York Heart Association (NYHA) Class ≥ II).

Sponsors & Collaborators

• Eastern Hepatobiliary Surgery Hospital: lead

Study Officials

• Feng Shen

  Eastern Hepatobiliary Surgery Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kui Wang

CONTACT

+8613636330827; wangkuiykl@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: January 10, 2026
• First Posted: January 20, 2026
• Study Start: January 30, 2026
• Primary Completion (Estimated): October 30, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share