Exosomal miRNA Liquid Biopsy for Early Liver Metastasis Detection in Pancreatic Cancer

NCT ID: NCT07224802

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

400 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-06-21

Study Completion Date

2026-06-18

Brief Summary

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Early liver metastasis (Early-LiM) is the most significant prognostic factor in pancreatic ductal adenocarcinoma (PDAC) and is thought to originate from occult micrometastases present at the time of surgery. Reliable preoperative detection of such lesions remains an unmet clinical need.

The EXELiM study aims to develop and validate a circulating exosomal microRNA (exo-miRNA)-based liquid biopsy assay to accurately identify PDAC patients at high risk of occult liver metastasis before surgery. By integrating machine learning with multi-institutional plasma exosome profiling, this study seeks to enable biology-driven patient stratification and guide treatment sequencing toward precision oncology.

Detailed Description

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Pancreatic ductal adenocarcinoma (PDAC) is one of the deadliest human malignancies, with a 5-year overall survival rate below 10%. Even after curative-intent pancreatectomy, over 70% of patients experience disease recurrence, with early liver metastasis (within six months postoperatively) representing the most aggressive and fatal pattern.

Emerging evidence indicates that pancreatic tumors release exosomes that modulate the liver microenvironment, establishing a premetastatic niche that facilitates early colonization. We hypothesize that circulating exosomal microRNAs reflect these biological processes and can serve as non-invasive biomarkers for detecting occult liver metastasis.

In this multi-center observational study, preoperative plasma-derived exosomes from PDAC patients are analyzed using next-generation small RNA sequencing and validated by RT-qPCR. A machine learning model is employed to integrate exo-miRNA expression profiles and clinical variables, yielding a predictive score for early liver metastasis risk.

The study evaluates the diagnostic accuracy, prognostic utility, and clinical benefit of the exo-miRNA panel compared to conventional biomarkers such as CA19-9.

Conditions

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PDAC - Pancreatic Ductal Adenocarcinoma

Keywords

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pancreatic ductal adenocarcinoma exosomal miRNA early liver metastasis liquid biopsy exosome

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

RETROSPECTIVE

Study Groups

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Training Cohort - PDAC with Occult Liver Metastasis

Patients with PDAC who were found to have occult liver metastases at the time of or shortly after curative-intent pancreatectomy (within 6 months post-surgery), in the first cohort (training set).

EXELiM assay (qRT-PCR validation)

Intervention Type DIAGNOSTIC_TEST

Quantitative reverse-transcription PCR (qRT-PCR)-based validation assay performed on preoperative plasma samples from PDAC patients in the training and validation cohorts.

Candidate microRNAs identified by small RNA sequencing were tested using the EXELiM assay to validate their predictive accuracy for occult liver metastasis detection prior to surgery.

Training Cohort - PDAC without Occult Liver Metastasis

Patients with PDAC who did not develop liver metastasis for at least 6 months after curative-intent surgery in the first cohort (training set).

EXELiM assay (qRT-PCR validation)

Intervention Type DIAGNOSTIC_TEST

Quantitative reverse-transcription PCR (qRT-PCR)-based validation assay performed on preoperative plasma samples from PDAC patients in the training and validation cohorts.

Candidate microRNAs identified by small RNA sequencing were tested using the EXELiM assay to validate their predictive accuracy for occult liver metastasis detection prior to surgery.

Validation Cohort - PDAC with Occult Liver Metastasis

Patients with PDAC who were found to have occult liver metastases (early postoperative hepatic recurrence within 6 months) in the second cohort (validation set).

EXELiM assay (qRT-PCR validation)

Intervention Type DIAGNOSTIC_TEST

Quantitative reverse-transcription PCR (qRT-PCR)-based validation assay performed on preoperative plasma samples from PDAC patients in the training and validation cohorts.

Candidate microRNAs identified by small RNA sequencing were tested using the EXELiM assay to validate their predictive accuracy for occult liver metastasis detection prior to surgery.

Validation Cohort - PDAC without Occult Liver Metastasis

Patients with PDAC who did not develop liver metastasis for at least 6 months after curative-intent surgery in the second cohort (validation set).

EXELiM assay (qRT-PCR validation)

Intervention Type DIAGNOSTIC_TEST

Quantitative reverse-transcription PCR (qRT-PCR)-based validation assay performed on preoperative plasma samples from PDAC patients in the training and validation cohorts.

Candidate microRNAs identified by small RNA sequencing were tested using the EXELiM assay to validate their predictive accuracy for occult liver metastasis detection prior to surgery.

Discovery Cohort - PDAC with Occult Liver Metastasis

Patients with PDAC who were found to have occult liver metastases at the time of or shortly after curative-intent pancreatectomy (within 6 months post-surgery), in the discovery cohort.

EXELiM small RNA sequencing

Intervention Type DIAGNOSTIC_TEST

High-throughput small RNA sequencing performed on preoperative plasma samples from PDAC patients in the discovery cohort to identify exosome-derived microRNAs associated with occult liver metastasis or early postoperative hepatic recurrence.

Discovery Cohort - PDAC without Occult Liver Metastasis

Patients with PDAC who did not develop liver metastasis for at least 6 months after curative-intent surgery in the discovery cohort.

EXELiM small RNA sequencing

Intervention Type DIAGNOSTIC_TEST

High-throughput small RNA sequencing performed on preoperative plasma samples from PDAC patients in the discovery cohort to identify exosome-derived microRNAs associated with occult liver metastasis or early postoperative hepatic recurrence.

Interventions

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EXELiM assay (qRT-PCR validation)

Quantitative reverse-transcription PCR (qRT-PCR)-based validation assay performed on preoperative plasma samples from PDAC patients in the training and validation cohorts.

Candidate microRNAs identified by small RNA sequencing were tested using the EXELiM assay to validate their predictive accuracy for occult liver metastasis detection prior to surgery.

Intervention Type DIAGNOSTIC_TEST

EXELiM small RNA sequencing

High-throughput small RNA sequencing performed on preoperative plasma samples from PDAC patients in the discovery cohort to identify exosome-derived microRNAs associated with occult liver metastasis or early postoperative hepatic recurrence.

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

* Histologically confirmed pancreatic ductal adenocarcinoma (PDAC)
* Undergoing curative-intent pancreatectomy
* Availability of preoperative plasma samples
* Complete clinical and follow-up data
* Written informed consent obtained

Exclusion Criteria

* Synchronous or secondary malignancies
* Non-adenocarcinoma histology
* Lack of informed consent
* Incomplete plasma sample data
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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City of Hope Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ajay Goel, PhD

Role: PRINCIPAL_INVESTIGATOR

City of Hope Medical Center

Locations

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City of Hope Medical Center

Duarte, California, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Ajay Goel, PhD

Role: CONTACT

Phone: 6263598111

Email: [email protected]

Facility Contacts

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Ajay Goel, PhD

Role: primary

References

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Other Identifiers

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19288/EXELiM

Identifier Type: -

Identifier Source: org_study_id