Dynamic Prediction Model for Patients with Unresectable Biliary Malignancies Receiving Systemic Chemotherapy Combined with Immunotherapy: a Multicenter, Observational Study (BTCICTY-001)

This study focused on the longitudinal tumor burden profile (tumor macro features, histopathological types and imaging features, etc.), liver function, health status, tumor biomarkers, and serological indicators of patients with unresectable biliary malignancies before chemotherapy combined with immunotherapy to build a dynamic prediction model. Based on this model, risk stratification of BTC patients was realized to explore which specific populations could safely initiate combination therapy. By constructing a risk stratification model, it can help clinicians to screen the best treatment population and provide a basis for safe treatment of high-risk patients.

Study Overview

• Status: Recruiting
• Conditions: Immunotherapy; Unresectable Biliary Tract Cancer; Dynamic Prediction Model
• Intervention / Treatment: Drug: Systemic chemotherapy combined with immunotherapy

• Study Type: Observational
• Enrollment (Estimated): 332

Contacts and Locations

• Study Contact: Name: Gao-Jun Teng, M.D; Phone Number: +86-02583272121; Email: gjteng@vip.sina.com

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China
      • Recruiting
      • Zhongda Hospital,
      • Contact: Gao-Jun Teng, M.D; Phone Number: +86-02583272121; Email: gjteng@vip.sina.com
    • Nanjing, Jiangsu, China
      • Active, not recruiting
      • Zhongda hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The target population of this project is patients diagnosed with unresectable biliary malignancies and treated with systemic chemotherapy combined with immunosuppressive agents.

Description

Inclusion Criteria:

• 1. Confirmed by histopathological diagnosis and/or clinical diagnosis (typical imaging features, clinical manifestations, laboratory examination, etc.) as biliary malignancy (Chinese Society of Clinical Oncology (CSCO) Guidelines for Diagnosis and Treatment of Biliary malignancy (2024)); 2. Based on unresectable malignant tumor in the diagnosis of biliary and in patients undergoing chemotherapy with immunosuppressant therapy system; 3. The liver function class for Child - Pugh, A or B; 4. More than 18 years of age, gender not limited; 5. Expected survival time for 3 months or more; 6. ECOG PS score 2 or less; 7. Meet the following laboratory test parameters:

  1. Hematological system function: Absolute neutrophil count ≥ 1.0×10⁹/L; platelet count ≥ 50×10⁹/L; hemoglobin ≥ 90 g/L; international normalized ratio less than 1.7 or prothrombin time prolongation not exceeding 4 seconds.
  2. Liver function: Alanine aminotransferase/aspartate aminotransferase not exceeding 5 times the upper limit of normal; total bilirubin ≤ 210 μmol/L [≤ 2.38 mg/dL]; albumin ≥ 28 g/L.
  3. Renal function: Serum creatinine not exceeding 1.5 times the upper limit of normal.

Exclusion Criteria:

• Malignant tumors other than BTC;
• Moderate to severe ascites (ascites reaching a Child-Pugh score of 3);
• Any local treatment (including transcatheter interventional therapy, ablation therapy, internal/external radiotherapy, etc.) or surgical resection or traditional Chinese medicine within 4 weeks before the combination of systemic chemotherapy and immunotherapy;
• Incomplete data, such as incomplete laboratory test data, missing or poor-quality imaging data, or lack of prognostic information;
• Severe liver dysfunction, such as decompensated cirrhosis and other liver diseases that significantly affect bilirubin levels;
• Severe comorbidities, such as refractory hypertension (blood pressure still higher than 150/100 mm Hg after optimal drug treatment), persistent arrhythmia (CTCAE grade 2 or above), any degree of atrial fibrillation, prolonged QTc interval (more than 450 milliseconds in men and more than 470 milliseconds in women), renal insufficiency, etc.;
• Human immunodeficiency virus (HIV) infection or acquired immune deficiency syndrome;
• Pregnant or lactating women;
• Acute or chronic mental disorders (including mental disorders that affect the subject's enrollment, treatment intervention, and follow-up).

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival(OS)	The OS is defined as the time from the initiation of any combination treatment to death due to any cause.	up to approximately 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse event(AE) per Common Terminology Criteria for Adverse Events(CTCAE) 5.0	The percentage and degree of patients who experience at least one AE, whether or not considered related to the treatment, according to CTCAE version 5.0.	up to approximately 2 years
Progression free survival(PFS) per Response Evaluation Criteria in Solid Tumors Version 1.1, modified Response Evaluation Criteria in Solid Tumors and immune-modified Response Evaluation Criteria In Solid Tumors(RECIST 1.1, mRECIST and iRECIST)	The PFS is defined as the time from the initiation of any combination treatment to the first documented progressive disease (according to RECIST 1.1, mRECIST and iRECIST) or death due to any cause, whichever occurs first.	up to approximately 2 years
Objective response rate(ORR) per Response Evaluation Criteria in Solid Tumors Version 1.1, modified Response Evaluation Criteria in Solid Tumors and immune-modified Response Evaluation Criteria In Solid Tumors(RECIST 1.1, mRECIST and iRECIST)	The ORR is defined as the proportion of patients with a documented complete response(CR) or partial response(PR) per RECIST 1.1, mRECIST and iRECIST.	up to approximately 2 years
Disease Control Rate (DCR) per Response Evaluation Criteria in Solid Tumors Version 1.1, modified Response Evaluation Criteria in Solid Tumors and immune-modified Response Evaluation Criteria In Solid Tumors(RECIST 1.1, mRECIST and iRECIST)	DCR is defined as the percentage of participants who have a best overall response of CR, PR, or stable disease (SD)per RECIST 1.1, mRECIST and iRECIST.	up to approximately 2 years

Collaborators and Investigators

• Sponsor: Zhongda Hospital
• Collaborators: Zhejiang Cancer Hospital; Shanghai Zhongshan Hospital; The First Affiliated Hospital of Zhengzhou University; Jiangsu Cancer Institute & Hospital; Eastern Hepatobiliary Surgery Hospital; Yunnan Cancer Hospital; The First Affiliated Hospital, University of Science and Technology of China; The Third Affiliated Hospital of Soochow University
• Investigators: Principal Investigator: Gao-jun Teng, M.D, Zhongda hospital, Southeast university, Nanjing, China

Study record dates

Study Major Dates

• Study Start (Actual): January 31, 2025
• Primary Completion (Estimated): March 31, 2026
• Study Completion (Estimated): May 31, 2026

Study Registration Dates

• First Submitted: February 23, 2025
• First Submitted That Met QC Criteria: February 23, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 23, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: immunotherapy; systemic chemotherapy; Dynamic prediction model; Unresectable Biliary Tract Cancer
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Biliary Tract Diseases; Biliary Tract Neoplasms; Immunologic Factors; Physiological Effects of Drugs; Immunomodulating Agents
• Other Study ID Numbers: 2024040196

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• product manufactured in and exported from the U.S.: No