Exploratory Study on the Therapeutic Effect Prediction Model of Advanced BTC Immunotherapy

Exploratory Study on the Therapeutic Effect Prediction Model of Immune Checkpoint Inhibitors for Advanced Biliary Tract Cancer

1. Establish a predictive model for the efficacy of immune checkpoint inhibitors (ICI) in Chinese patients with biliary tract cancers. By analyzing the dynamic changes of circulating tumor DNA (ctDNA) and other clinical and pathological features before and after ICI treatment in a cohort of patients with biliary tract tumors, a predictive model can be established to evaluate the efficacy of ICI treatment in the early stages or even before treatment, serving as a reliable tool for selecting patients who are likely to benefit from ICI treatment.
2. Investigate the clinical features of populations that benefit from different immune combination therapies. By comparing the differences and enrichment of mutations between patients receiving different treatment regimens, and if patients have sufficient pre-treatment tissue, further comparisons of differentially expressed genes and pathways may be made.

Study Overview

• Status: Active, not recruiting
• Conditions: Biliary Tract Cancer; Predictive Cancer Model; Immune Checkpoint Inhibitor
• Intervention / Treatment: Drug: Immune Checkpoint Inhibitors

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100730
      • Chinese Academy of Medical Sciences & Peking Union Medical College Hospital (CAMS&PUMCH)

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• The subjects voluntarily participate in the study and agree to sign the informed consent form, are compliant, and cooperate with follow-up.
• Patients with advanced biliary tract cancers (including gallbladder cancer, intrahepatic cholangiocarcinoma (ICC), hilar cholangiocarcinoma, or distal cholangiocarcinoma) with histologically, cytologically, or clinically confirmed.
• Patients who have undergone curative surgery and experienced disease recurrence after more than 6 months; or patients who have completed adjuvant therapy (chemotherapy and/or radiotherapy) and have been disease-free for more than 6 months after completing adjuvant therapy are eligible for inclusion.
• Regardless of gender, age 18-80 years old.
• ECOG PS of 0-2 with expected survival time ≥12 weeks.
• According to RECIST V1.1, the patient had at least one measurable lesion.
• The patient is suitable for treatment with immune checkpoint inhibitors (PD-1/PD-L1 inhibitor).

Exclusion Criteria:

• Suffering from other malignant tumors.
• Previous systemic treatment received.
• Patients with serious organic diseases cannot receive the treatment designed in this study;
• Having a psychiatric disorder does not guarantee compliance with this study.
• Patients with symptomatic brain metastasis or complications related to brain metastasis or cognitive impairment.
• Adverse events (except hair loss of any grade) from previous antitumor therapy did not return to ≤ grade 1 or better (According to CTCAE version 5.0).
• Previously received any cell or organ transplant treatment.
• Allergy to the therapeutic drugs of this study or CT or MRI-enhanced contrast agents.
• Received local hepatobiliary treatment (including various types of ablations, percutaneous ethanol or acetic acid injection, high-intensity focused ultrasound, transarterial embolization, chemotherapy, or chemoembolization) within 14 days before the start of the study treatment.
• The physician determines that there are any contraindications to ICI treatment.
• During the second blood monitoring, the disease changes could not be judged (such as unexplained ascites, worsening symptoms); Or non-disease progression, but the next immunotherapy is not planned.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Immunotherapy cohort; Patients who were administered with immunotherapy	Drug: Immune Checkpoint Inhibitors; Immune Checkpoint Inhibitors including Durvalumab, Pembrolizumab and Toripalimab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression free survival	It refers to the time from the first administration of the regimen to the first occurrence of disease progression or death due to any cause in the subjects.	Baseline up to approximately 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate	The proportion of patients whose tumor volume shrinks to a predetermined value and can maintain the minimum required duration, including complete response and partial response.	Baseline up to approximately 6 months
Overall survival	The time from randomization to death due to any cause in the subjects.	Baseline up to approximately 15 months
Time to treatment discontinuation	The time from the beginning of ICI drug use to the cessation of ICI drug use for any reason	Baseline up to approximately 12 months

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Collaborators: Geneplus-Beijing Co. Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2021
• Primary Completion (Actual): December 30, 2023
• Study Completion (Estimated): August 1, 2024

Study Registration Dates

• First Submitted: September 24, 2023
• First Submitted That Met QC Criteria: October 5, 2023
• First Posted (Actual): October 10, 2023

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 7, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms; Neoplasms by Site; Digestive System Neoplasms; Biliary Tract Diseases; Biliary Tract Neoplasms; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Antineoplastic Agents, Immunological; Immune Checkpoint Inhibitors
• Other Study ID Numbers: JYJ-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No