Advanced Biliary Tract Cancer(BTC)Phase 2NCT07351591
A Phase II Trial of Organoid Drug Sensitivity Testing to Guide Therapy in Unresectable Biliary Tract Cancers
A Phase 2 study testing Organoid Drug Sensitivity Testing-Guided Therapy in people with Advanced Biliary Tract Cancer(BTC), run by Eastern Hepatobiliary Surgery Hospital.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Advanced Biliary Tract Cancer(BTC)
- What is being tested?
- Organoid Drug Sensitivity Testing-Guided Therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 88 participants
- Where
- No sites listed yet
- Study dates
- 30 Jan 2026to 31 Dec 2027
- Phase
- Phase 2
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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):
- 9.1. 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
- Serum Albumin ≥ 28 g/L 9.3. Renal:
- Serum Creatinine (Cr) ≤ 1.5 × ULN or Creatinine Clearance (CCr) ≥ 50 mL/min (calculated using the Cockcroft-Gault formula)
- Urinalysis indicates urine protein < 2+; if baseline urinalysis shows urine protein ≥ 2+, a 24-hour urine collection must demonstrate 24-hour urine protein quantification < 1 g.
- 9.4. Coagulation:
- International Normalized Ratio (INR) and Activated Partial Thromboplastin Time (APTT) ≤ 1.5 × ULN
- For women of childbearing potential (WOCBP), a negative urine or serum pregnancy test must be confirmed within 3 days prior to the first dose of study drug (Cycle 1, Day 1). If a urine test is inconclusive, a serum pregnancy test is required. WOCBP and male subjects must agree to use adequate contraception during the observation period and for at least 8 weeks after the last dose of the study drug. A woman is considered not of childbearing potential if she is postmenopausal (≥1 year without menses) or has undergone surgical sterilization (bilateral oophorectomy, hysterectomy, or bilateral tubal ligation). Subjects (both male and female) with risk of pregnancy must use highly effective contraception (with a failure rate of <1% per year) during the entire treatment period and for 120 days after the last dose of the study drug (or 180 days after the last dose of chemotherapy agents).
Who cannot join
- 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:
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- 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).
- Myocardial infarction within 6 months prior to randomization.
- History of esophageal or gastric variceal bleeding within 6 months prior to enrollment.
- Poorly controlled hypertension (systolic blood pressure >140 mmHg and/or diastolic blood pressure >90 mmHg).
- History of non-infectious pneumonitis requiring corticosteroid treatment within 1 year prior to the first dose, or current clinically active interstitial lung disease.
- Active tuberculosis.
- Active or uncontrolled infection requiring systemic therapy.
- Clinically active diverticulitis, intra-abdominal abscess, or gastrointestinal obstruction.
- Decompensated liver disease, acute or chronic active hepatitis.
- Unstable or active peptic ulcer disease, or patients with gastrointestinal bleeding.
- Poorly controlled diabetes mellitus (fasting blood glucose >10 mmol/L).
- Urinalysis indicating urine protein ≥ ++ and confirmed 24-hour urine protein quantification >1.0 g.
- Uncontrolled hypercalcemia (>1.5 mmol/L ionized calcium, or calcium >12 mg/dL, or corrected serum calcium > ULN), or symptomatic hypercalcemia requiring continued bisphosphonate therapy.
- Non-healing wound(s) or fracture(s).
- Psychiatric disorder that compromises the ability to comply with the treatment protocol.
- Female subjects who are pregnant or breastfeeding.
- Assessed by the investigator as being unable or unwilling to comply with the requirements of the study protocol.
- Known allergy to any of the study drug(s) used in this 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: Patient-Derived Tumor Organoid Drug Sensitivity Testing to Guide Treatment Selection in Patients With Unresectable Biliary Tract Cancers: A Prospective, Non-randomized, Open-label, Single-Center Phase II Clinical Trial
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.
Read the detailed description
- Study Design: This is a single-center, prospective, non-randomized, open-label, controlled phase II clinical trial conducted at the Third Affiliated Hospital of Naval Medical University.
- Study Population: The study will enroll 88 adult patients (aged 18-70) with unresectable biliary tract cancers, confirmed per NCCN guidelines. Participants must have an ECOG performance status of 0-1, a life expectancy of >12 weeks, and adequate organ function.
- Interventions: Organoid Drug Sensitivity Testing (ODST) Group: Tumor tissue from biopsies is used to establish patient-derived organoids. Successful organoids undergo drug sensitivity testing against a predefined panel of regimens. The most effective drug(s), based on IC50 and AUC values, are recommended for treatment. Control Group (Standard Therapy): Patients receive physician's choice of standard systemic therapy, including GC, GEMOX, or other approved regimens.
- Study Endpoints: Primary Endpoints: Objective Response Rate (ORR) as assessed by RECIST 1.1. Progression-Free Survival (PFS). Secondary Endpoints: Overall Survival (OS). Safety and tolerability, assessed by the incidence and severity of adverse events (AEs, SAEs) according to NCI CTCAE v5.0.
- Follow-up: Patients will be followed regularly with imaging assessments every 42 days (±3 days) for the first 48 weeks, and every 63 days (±3 days) thereafter. Laboratory tests and clinical evaluations will be conducted to monitor treatment response and adverse events until death, withdrawal of consent, or study termination.
- Statistical Analysis:
Sample size was calculated based on an assumed improvement in ORR from 26.7% in the control group to 66.7% in the ODST-guided group (α=0.05, power=80%, 1:1 allocation, 10% dropout rate). Statistical analyses will be performed using SAS, with descriptive statistics for safety and efficacy analyses.
- Conditions
- Advanced Biliary Tract Cancer(BTC)
- Being tested
- Organoid Drug Sensitivity Testing-Guided Therapy
- Keywords
- organoid, drug-sensitive test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Organoid Drug Sensitivity Testing-Guided TherapyDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Physician's Choice Group：According to empirical and clinical practice Receives: Drug: Organoid Drug Sensitivity Testing-Guided Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate(ORR)
ORR was assessed by investigators per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1).
Measured: 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.
Measured: up to 3 years
Secondary outcomes (2)
Overall Survival(OS)
Measured: Up to 3 years
Adverse event
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2026
- Study start30 Jan 2026 (estimated)
- Primary completion30 Oct 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Biliary Tract Cancer(BTC)Studies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eastern Hepatobiliary Surgery Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shen Feng, Eastern Hepatobiliary Surgery Hospital
- Investigators
- Feng Shen, Principal investigator, Eastern Hepatobiliary Surgery Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07351591
- Sponsor's protocol ID
- EHBHKY2025-H033-P001
- Status last verified
- Jan 2026
- First submitted
- 10 Jan 2026
- Last updated
- 20 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient-Derived Tumor Organoid Drug Sensitivity Testing to Guide Treatment Selection in Patients With Unresectable Biliary Tract Cancers: A Prospective, Non-randomized, Open-label, Single-Center Phase II Clinical Trial
- ClinicalTrials.gov ID
- NCT07351591
- Acronym / sponsor ID
- EHBHKY2025-H033-P001
- Conditions
- Advanced Biliary Tract Cancer(BTC)
- Keywords
- organoid, drug-sensitive test
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- Eastern Hepatobiliary Surgery HospitalUniversity, hospital or other
- Study start
- 30 Jan 2026 (estimated)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 20 Jan 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.
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.
Detailed description
- Study Design: This is a single-center, prospective, non-randomized, open-label, controlled phase II clinical trial conducted at the Third Affiliated Hospital of Naval Medical University.
- Study Population: The study will enroll 88 adult patients (aged 18-70) with unresectable biliary tract cancers, confirmed per NCCN guidelines. Participants must have an ECOG performance status of 0-1, a life expectancy of >12 weeks, and adequate organ function.
- Interventions: Organoid Drug Sensitivity Testing (ODST) Group: Tumor tissue from biopsies is used to establish patient-derived organoids. Successful organoids undergo drug sensitivity testing against a predefined panel of regimens. The most effective drug(s), based on IC50 and AUC values, are recommended for treatment. Control Group (Standard Therapy): Patients receive physician's choice of standard systemic therapy, including GC, GEMOX, or other approved regimens.
- Study Endpoints: Primary Endpoints: Objective Response Rate (ORR) as assessed by RECIST 1.1. Progression-Free Survival (PFS). Secondary Endpoints: Overall Survival (OS). Safety and tolerability, assessed by the incidence and severity of adverse events (AEs, SAEs) according to NCI CTCAE v5.0.
- Follow-up: Patients will be followed regularly with imaging assessments every 42 days (±3 days) for the first 48 weeks, and every 63 days (±3 days) thereafter. Laboratory tests and clinical evaluations will be conducted to monitor treatment response and adverse events until death, withdrawal of consent, or study termination.
- Statistical Analysis:
Sample size was calculated based on an assumed improvement in ORR from 26.7% in the control group to 66.7% in the ODST-guided group (α=0.05, power=80%, 1:1 allocation, 10% dropout rate). Statistical analyses will be performed using SAS, with descriptive statistics for safety and efficacy analyses.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Organoid Drug Sensitivity Testing-Guided TherapyPhysician's Choice Group：According to empirical and clinical practice |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- 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.
Key exclusion criteria (32 listed)
- 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.
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 |
|---|---|---|
| Organoid Drug Sensitivity Testing-Guided TherapyFresh 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 | Drug | Arm 1 |
Primary outcome
Objective Response Rate(ORR)
Time frame: Through out the study (up to 3 years)
ORR was assessed by investigators per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1).
Progression-free Survival(PFS)
Time frame: up to 3 years
In treated subjects, the time from first recording to tumor progression (based on RECIST 1.1 and mRECIST) or death from any cause.
2 secondary outcomes
Overall Survival(OS)
Time frame: Up to 3 years
Adverse event
Time frame: 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.
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 20 Jan 2026.
- Individual participant dataSharing planned: No
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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.
Feng Shen principal investigator
Eastern Hepatobiliary Surgery Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Organoid Drug Sensitivity Testing-Guided Therapy in people with Advanced Biliary Tract Cancer(BTC), run by Eastern Hepatobiliary Surgery 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 21 inclusion and 32 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 20 Jan 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Kui Wang (+8613636330827, wangkuiykl@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate(ORR) (time frame: Through out the study (up to 3 years)). 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 20 Jan 2026.