Prediction of Neoadjuvant Chemotherapy Response in Pancreatic Cancer (PRECEPT)

November 18, 2025 updated by: City of Hope Medical Center

An Exosomal miRNA Based Predictive Model for Personalized Neoadjuvant Chemotherapy Selection in Pancreatic Ductal Adenocarcinoma

This study aims to develop and validate a predictive microRNA (miRNA) panel to assess the response to neoadjuvant chemotherapy (NACT) in patients with resectable and borderline resectable pancreatic ductal adenocarcinoma (PDAC).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal malignancies, with a five-year overall survival rate below 12%. Surgical resection combined with systemic chemotherapy offers the best chance for cure; however, only a subset of patients truly benefits from neoadjuvant chemotherapy (NACT). Currently, there are no validated biomarkers to predict response to NACT, making treatment selection largely empirical.

The PRECEPT study (PREdiction of Chemotherapy Effect in Pancreatic Cancer Treatment) aims to identify and validate microRNA (miRNA)-based biomarkers from pre-treatment plasma that can predict therapeutic response to neoadjuvant chemotherapy in patients with resectable or borderline resectable PDAC. Specifically, the study focuses on two standard regimens: FOLFIRINOX and gemcitabine plus nab-paclitaxel (GEM-NABP).

This is a retrospective, non-interventional, observational study using archived plasma samples collected before the initiation of NACT. Exosomal miRNA sequencing (small RNA-seq) has been performed to identify candidate predictive miRNAs. These candidates will be validated using quantitative reverse transcription PCR (qRT-PCR) in an independent patient cohort. The association between miRNA expression levels and pathologic response (CAP grade or tumor regression score) will be analyzed. Additionally, correlations with overall survival (OS) and recurrence-free survival (RFS) will be explored.

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Ajay Goel, PhD
  • Phone Number: 626-218-3452
  • Email: AJGOEL@COH.ORG

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91016
        • Recruiting
        • City of Hope Medical Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients diagnosed with resectable or borderline resectable pancreatic ductal adenocarcinoma (PDAC) who received neoadjuvant chemotherapy (FOLFIRINOX or Gemcitabine + Nab-paclitaxel) followed by surgery.

Description

Inclusion Criteria:

  • Histologically confirmed pancreatic ductal adenocarcinoma (PDAC).
  • Underwent neoadjuvant chemotherapy (FOLFIRINOX or Gemcitabine/nab-paclitaxel).
  • Availability of pre-treatment plasma samples.
  • Underwent curative-intent resection (R0 or R1).

Exclusion Criteria:

  • Inadequate plasma samples or poor RNA quality for exosomal miRNA analysis.
  • Non-adenocarcinoma histology.
  • Presence of synchronous or multiple primary malignancies.
  • Receipt of chemotherapy regimens other than standard FOLFIRINOX or gemcitabine plus nab-paclitaxel (GEM-NABP).
  • Presence of active inflammatory or autoimmune diseases.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Discovery Cohort - NAC Responder Group

Patients with resectable or borderline resectable pancreatic ductal adenocarcinoma (PDAC) who received neoadjuvant chemotherapy (FOLFIRINOX or gemcitabine plus nab-paclitaxel) and achieved a clinical or pathological response.

Responders were defined as patients showing complete response (CR), partial response (PR), or stable disease (SD) according to radiologic or pathologic assessment after NACT.

Pre-treatment plasma samples from these patients were analyzed by small RNA sequencing to identify microRNAs associated with favorable chemotherapy response.
Diagnostic test: Small RNA sequencing
High-throughput small RNA sequencing performed on pre-treatment plasma samples from PDAC patients in the Discovery cohort to identify candidate microRNAs associated with neoadjuvant chemotherapy response. Sequencing data were analyzed to detect differentially expressed miRNAs between responder (CR + PR + SD) and non-responder (PD) groups.
Discovery Cohort - NAC Non-Responder Group

Patients with resectable or borderline resectable PDAC who received neoadjuvant chemotherapy but demonstrated progressive disease (PD) on radiologic or pathologic evaluation.

Pre-treatment plasma samples from these patients were analyzed in parallel by small RNA sequencing to identify differential miRNA expression compared with responders.
Diagnostic test: Small RNA sequencing
High-throughput small RNA sequencing performed on pre-treatment plasma samples from PDAC patients in the Discovery cohort to identify candidate microRNAs associated with neoadjuvant chemotherapy response. Sequencing data were analyzed to detect differentially expressed miRNAs between responder (CR + PR + SD) and non-responder (PD) groups.
Training Cohort - NAC Responder Group

PDAC patients treated with neoadjuvant chemotherapy (FOLFIRINOX or gemcitabine plus nab-paclitaxel) who achieved CR, PR, or SD responses.

Candidate microRNAs identified in the Discovery cohort were validated using qRT-PCR (PRECEPT assay).

Responder group data were used to train and optimize the predictive miRNA panel.
Diagnostic test: PRECEPT assay (qRT-PCR validation)
Quantitative reverse transcription PCR (qRT-PCR)-based validation assay performed on pre-treatment plasma samples in the Training and Validation cohorts. Candidate microRNAs identified in the Discovery cohort by small RNA sequencing were tested using the PRECEPT assay to develop and validate a predictive miRNA panel for neoadjuvant chemotherapy response.
Training Cohort - NAC Non-Responder Group

PDAC patients who received neoadjuvant chemotherapy but exhibited progressive disease (PD).

Plasma miRNA expression was measured using the PRECEPT assay and compared with responders to refine the predictive model for chemotherapy response.
Diagnostic test: PRECEPT assay (qRT-PCR validation)
Quantitative reverse transcription PCR (qRT-PCR)-based validation assay performed on pre-treatment plasma samples in the Training and Validation cohorts. Candidate microRNAs identified in the Discovery cohort by small RNA sequencing were tested using the PRECEPT assay to develop and validate a predictive miRNA panel for neoadjuvant chemotherapy response.
Validation Cohort - NAC Responder Group

A separate validation cohort of PDAC patients treated with neoadjuvant chemotherapy who achieved CR, PR, or SD.

The established PRECEPT miRNA panel (qRT-PCR) was applied to evaluate predictive accuracy in this independent responder group.
Diagnostic test: PRECEPT assay (qRT-PCR validation)
Quantitative reverse transcription PCR (qRT-PCR)-based validation assay performed on pre-treatment plasma samples in the Training and Validation cohorts. Candidate microRNAs identified in the Discovery cohort by small RNA sequencing were tested using the PRECEPT assay to develop and validate a predictive miRNA panel for neoadjuvant chemotherapy response.
Validation Cohort - NAC Non-Responder Group

Independent PDAC patients who received neoadjuvant chemotherapy and demonstrated progressive disease (PD).

This cohort was used to confirm the predictive performance and robustness of the PRECEPT miRNA assay compared with responders.
Diagnostic test: PRECEPT assay (qRT-PCR validation)
Quantitative reverse transcription PCR (qRT-PCR)-based validation assay performed on pre-treatment plasma samples in the Training and Validation cohorts. Candidate microRNAs identified in the Discovery cohort by small RNA sequencing were tested using the PRECEPT assay to develop and validate a predictive miRNA panel for neoadjuvant chemotherapy response.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pathological Response Rate
Time Frame: up to 1 year
Proportion of patients achieving partial or complete pathologic response after neoadjuvant chemotherapy, as assessed using resected pancreatic cancer specimens.
up to 1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Recurrence-Free Survival (RFS)
Time Frame: Up to 3 years after surgery
Time from date of surgical resection to first documented recurrence or death from any cause, whichever occurs first.
Up to 3 years after surgery
Overall Survival (OS)
Time Frame: Up to 5 years after surgery
Time from date of surgical resection to date of death from any cause. Patients alive at last follow-up will be censored.
Up to 5 years after surgery
Radiologic Response Rate
Time Frame: up to 1 year
Proportion of patients achieving partial or complete radiologic response during neoadjuvant chemotherapy, as assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 using contrast-enhanced CT or MRI scans.
up to 1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

City of Hope Medical Center

Investigators

  • Principal Investigator: Ajay Goel, PhD, City of Hope Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 15, 2024

Primary Completion (Estimated)

June 18, 2026

Study Completion (Estimated)

June 18, 2026

Study Registration Dates

First Submitted

November 3, 2025

First Submitted That Met QC Criteria

November 5, 2025

First Posted (Estimated)

November 7, 2025

Study Record Updates

Last Update Posted (Actual)

November 21, 2025

Last Update Submitted That Met QC Criteria

November 18, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • 19288/PRECEPT

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Data collected for the study will be made available to others, including de-identified participant data, at publication, via a signed data access agreement and at the discretion of the investigators' approval of the proposed use of such data.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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