Validation of the Proteomic Profiling as a Diagnostic Test for Extra-hepatic Cholangiocarcinoma (PROFI-CHOL)

Proteomic Profiling as a Diagnostic Test for Extrahepatic Cholangiocarcinoma on Cytological Samples : Validation of a Proof-of-concept.

The study aims to valide a proof of a concept of the proteomic profiling as a diagnostic tool for bile duct stenosis suspicious of cholangiocarcinoma. The main objective is to evaluate the addition of proteomic profiling to the conventional histological diagnosis of endo-biliary cytological sampling of biliary stenosis, compared with cytological sampling alone. With the addition of proteomic profiling to the conventional histological diagnostic technique, an overall diagnostic sensitivity of 80% is expected

Study Overview

• Status: Not yet recruiting
• Conditions: Cholangiocarcinoma
• Intervention / Treatment: Diagnostic test: Proteomic profiling

Detailed Description

Cholangiocarcinoma is a cancer with an increasing incidence and a poor prognosis (< 5% of all stages at 5 years). The extra-hepatic form is the most common and is rarely resectable at diagnosis (30% of cases). This is due to the late onset of symptoms, which often indicate that the disease is too advanced. It can also be explained by the diagnostic difficulties encountered with this cancer. In fact, the reference diagnostic technique is histological testing on endo-biliary cytological samples taken by biopsy and/or brushing during catheterisation of the bile ducts or by interventional radiology. The problem is that the sensitivity of this test is of the order of 50%, with an additional diagnostic uncertainty of the order of 20 to 30%, due to cellular atypia that are not sufficiently clear to confirm a cancerous pathology, although it is not possible to exclude it. This leads to diagnostic error and delay, repeated invasive examinations and sometimes major surgery for stenoses that are ultimately benign when the surgical decision has been taken without a reliable histological result. Additional diagnostic techniques are needed to diagnose a suspected extrahepatic cholangiocarcinoma stenosis, particularly at the molecular level using proteomics and, above all, proteomic profiling. Proteomic profiling enables a patient's diagnosis to be oriented towards a profile (benign or malignant) by comparing the proteins identified in formalin-fixed, paraffin-embedded tissue (cytological sample) with a set of proteins identified within a reference diagnostic signature obtained from previously analysed benign and malignant samples. Within Inserm Unit 1312 - Team 3, a proof of concept for proteomic profiling has been developed as a diagnostic tool in the face of suspected extrahepatic cholangiocarcinoma stenosis, with the development of a diagnostic signature. The aim of this project is to validate this proof of concept on a large retrospective monocentric cohort.

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

Study Locations

• France
  • Pessac, France
    • CHU Bordeaux
    • Contact: Arthur Marichez, MD; Phone Number: +335 57 65 60 02; Email: arthur.marichez@chu-bordeaux.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Patient with biliary stenosis of undetermined origin who has had an endo-biliary cytological sample (biopsy and/or brushing) for suspected cholangiocarcinoma

Description

Inclusion Criteria:

• Age ≥ 18 years
• Patient with biliary stenosis of undetermined origin who has had an endo-biliary cytological sample (biopsy and/or brushing) for suspected cholangiocarcinoma, fixed in formalin and included in paraffin with at least 1 year of follow-up.
• No objection to re-use of data

Exclusion Criteria:

• Absence of cellular material usable in proteomics on residual histological block
• Presence of pancreatic adenocarcinoma demonstrated on cytological sampling, definitive histology after surgical resection and/or clinical-morphological follow-up of at least one year.
• Patient under legal protection

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Suspected cholangiocarcinoma; Patient with biliary stenosis of undetermined origin who has had an endo-biliary cytological sample (biopsy and/or brushing) for suspected cholangiocarcinoma	Diagnostic test: Proteomic profiling; Proteomic profiling as a diagnostic tool in the face of suspected extrahepatic cholangiocarcinoma stenosis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sensitivity of the proteomic profiling	Sensitivity of the proteomic profiling test combined with conventional histological diagnosis, compared with the sensitivity of the conventional histological diagnostic test alone.	Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Accuracy of the proteomic profiling	The accuracy of the proteomic profiling associated with the conventional histological test (specificity, positive predictive value, negative predictive value)	Baseline
Molecular pathway of cholangiocarcinogenesis	Identification of the molecular pathway of cholangiocarcinogenesis using Gene Set Enrichment Analysis	Baseline

Collaborators and Investigators

• Sponsor: University Hospital, Bordeaux
• Collaborators: University of Bordeaux; Plateforme Oncoprot (TBMCore 3427 - US005, Bordeaux Biologie Santé)

Study record dates

Study Major Dates

• Study Start (Estimated): November 1, 2025
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): March 1, 2026

Study Registration Dates

• First Submitted: January 13, 2025
• First Submitted That Met QC Criteria: January 13, 2025
• First Posted (Actual): January 17, 2025

Study Record Updates

• Last Update Posted (Actual): June 29, 2025
• Last Update Submitted That Met QC Criteria: June 27, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: proteomic; cholangiocarcinoma; diagnosis; biopsy; proteomic profiling
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Carcinoma; Cholangiocarcinoma
• Other Study ID Numbers: CHUBX 2024/32

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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