Patient-Derived Organoid-on-a-Chip Model for Personalized Prediction of Chemotherapy Efficacy in Pancreatic Cancer

NCT07683845 | Recruiting

• 1 other identifier: DX-PDAC-01
• Study Type: observational
• Target: 164
• Locations: 1 country
• Sites: 1

Brief Summary

1. 1.Background Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal malignancies worldwide, with a 5-year survival rate below 10%. Systemic chemotherapy is the mainstay of treatment for the majority of patients who present with unresectable disease. However, the objective response rates for standard first-line regimens, such as nab-paclitaxel plus gemcitabine (AG) and FOLFIRINOX, are only 15-30%. Currently, there are no validated predictive biomarkers to guide chemotherapy selection, leading to a trial-and-error approach that exposes patients to unnecessary toxicity and delays effective treatment.
2. 2.Study Objective The primary objective of this study is to evaluate the concordance between PDO-based drug sensitivity test results and clinical efficacy in PDAC patients receiving standard first-line chemotherapy. Secondary objectives include optimizing culture conditions and drug testing protocols for pancreatic cancer PDOs derived from surgical and biopsy specimens.
3. 3.Study Design This is a prospective, observational, multicenter cohort study. The study will enroll treatment-naïve patients aged 18-75 years with histologically confirmed PDAC who are scheduled to receive first-line chemotherapy. Fresh tumor tissue will be obtained from routine surgical resection or biopsy procedures. PDOs will be established from the collected specimens and subjected to in vitro drug sensitivity testing against the corresponding chemotherapy regimens. The testing results will be classified as "sensitive" or "insensitive" based on IC50 values and maximum inhibition rates.
4. 4.Study Population Approximately 164 patients will be screened to achieve 80 evaluable cases, accounting for a PDO culture success rate of 85%, a follow-up rate of 90%, and exclusions due to non-PDAC pathology or non-first-line treatment regimens. Eligible patients must have adequate organ and bone marrow function, an ECOG performance status of 0-2, and a life expectancy of at least 3 months. Exclusion criteria include inadequate sample quality, PDO culture failure, pregnancy, severe comorbidities, and unstable medical conditions.
5. 5.Study Sites This multicenter study is being conducted at six tertiary medical centers in China: Ruijin Hospital (Shanghai, lead site), Peking University Cancer Hospital, Beijing Friendship Hospital, The Second Hospital of Tianjin Medical University, The Second Xiangya Hospital of Central South University, and Shenzhen People's Hospital.
6. 6.Risks and Benefits This observational study poses no additional physical risk to participants, as samples are collected from residual tissue during routine diagnostic or therapeutic procedures. No investigational interventions are administered. Participants will not receive direct medical benefit from study participation; however, the findings may contribute to the development of a clinically applicable predictive tool to guide chemotherapy selection for future pancreatic cancer patients, potentially improving treatment outcomes and quality of life.
7. 7.Ethical Considerations The study will be conducted in accordance with the Declaration of Helsinki and applicable Chinese regulations. The protocol has been reviewed and approved by the institutional ethics committee. Written informed consent will be obtained from all participants prior to enrollment. All data will be anonymized and handled in strict compliance with privacy protection regulations.
8. 8.Timeline The study is planned from February 2026 to February 2028.

Conditions

Pancreatic Ductal Adenocarcinoma (PDAC)

Keywords

Pancreatic Ductal Adenocarcinoma; Patient-Derived Organoids; Drug Sensitivity Testing; Precision Medicine; Chemotherapy Response Prediction

Outcome Measures

Primary Outcomes (1)

• Concordance Between Organoid Drug Sensitivity Test Results and Clinical Chemotherapy Efficacy in Pancreatic Ductal Adenocarcinoma Patients, Assessed by Sensitivity, Specificity, and Overall Accuracy

  Concordance between organoid drug sensitivity test results and clinical chemotherapy efficacy in pancreatic ductal adenocarcinoma patients. Organoid sensitivity is determined by exposing patient-derived organoids to chemotherapy regimens across 5-7 concentrations, measuring cell viability via ATP content, generating concentration-inhibition curves, and deriving IC50 values, with results classified as "sensitive" or "insensitive." Clinical efficacy is assessed using RECIST v1.1 criteria after six treatment cycles, classifying outcomes as complete response, partial response, stable disease (with CA199 decrease or SDa) as effective, and stable disease (with CA199 increase or SDb) or progressive disease as ineffective. Concordance is calculated as sensitivity, specificity, and overall accuracy using a four-fold table with 95% confidence intervals. Unit of Measure: Percentage (%) - specifically, sensitivity (%), specificity (%), and overall accuracy (%).

  At the end of Cycle 6 (each cycle is 28 days), or at pre-surgery imaging for neoadjuvant patients; if death occurs before first post-treatment imaging, classified as ineffective.

Secondary Outcomes (3)

• Objective Response Rate (ORR) in Organoid-Sensitive vs. Insensitive Groups

  Assessed at the end of Cycle 6 (each cycle is 28 days). For neoadjuvant patients, assessed at pre-surgery imaging. If death occurs before first post-treatment imaging, classified as non-responder.

• Disease Control Rate (DCR) in Organoid-Sensitive vs. Insensitive Groups

  Assessed at the end of Cycle 6 (each cycle is 28 days). For neoadjuvant patients, assessed at pre-surgery imaging. If death occurs before first post-treatment imaging, classified as non-responder.

• Progression-Free Survival (PFS) in Organoid-Sensitive vs. Insensitive Groups

  Assessed every 3 treatment cycles (each cycle is 28 days) from treatment initiation until disease progression or death from any cause, with primary analysis time points at 6 months and 12 months post-treatment initiation.

Study Arms (1)

Pancreatic Cancer Patients Receiving First-Line Chemotherapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population comprises treatment-naïve patients aged 18-75 years with histologically confirmed pancreatic ductal adenocarcinoma (PDAC) scheduled to receive first-line chemotherapy for borderline resectable, locally advanced, or metastatic disease. Eligible patients must have measurable disease per RECIST 1.1, ECOG performance status 0-2, life expectancy ≥3 months, and adequate organ function. Patients must provide sufficient fresh tumor tissue from surgery or biopsy (EUS-FNB 19G/22G) with tumor cell content and viability \>50%. Approximately 164 patients will be screened to achieve 80 evaluable cases, accounting for follow-up rate of 90%, organoid culture success rate of 85%, and exclusions due to non-PDAC pathology or non-first-line treatment. All participants must sign informed consent and comply with follow-up.

You may qualify if:

• Age 18-75 years, both sexes
• Pathologically confirmed pancreatic ductal adenocarcinoma (PDAC) in treatment-naïve patients
• Ability to obtain sufficient fresh tumor tissue samples for organoid establishment through surgery or biopsy
• Planned to receive neoadjuvant therapy for borderline resectable disease, conversion therapy for locally advanced disease, or first-line therapy for metastatic disease, using single-agent or combination chemotherapy
• At least one measurable lesion per RECIST 1.1 criteria
• ECOG performance status 0-2
• Life expectancy ≥3 months
• Adequate organ and bone marrow function: hemoglobin ≥9.0 g/dL; absolute neutrophil count ≥1.5×10⁹/L; platelet count ≥100×10⁹/L; total bilirubin ≤1.5×ULN (or ≤3×ULN with biliary obstruction); ALT/AST ≤2.5×ULN (or ≤5×ULN with biliary obstruction); serum creatinine ≤1.5×ULN or CrCl \>60 mL/min
• Willing to sign informed consent, good compliance, and able to comply with follow-up

You may not qualify if:

• Sample quantity, quality, or preservation does not meet requirements
• Expected transport time \>24 hours (including samples stored overnight before delivery)
• Organoid culture failure or testing quality control failure
• Inability to tolerate chemotherapy regimens
• Severe comorbidities, uncontrolled conditions, or active infections
• Pregnancy or breastfeeding
• History of severe autoimmune diseases
• Any unstable condition that may compromise patient safety or compliance

Sponsors & Collaborators

• Beijing Daxiang Biotech Co., Ltd: lead
• Ruijin Hospital: collaborator
• Tianjin Medical University Second Hospital: collaborator
• Second Xiangya Hospital of Central South University: collaborator
• Shenzhen People's Hospital: collaborator
• Beijing Friendship Hospital: collaborator
• Peking University Cancer Hospital & Institute: collaborator

Study Sites (1)

Ruijin Hospital

Shanghai, Shanghai Municipality, 200025, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Fresh tumor tissue samples will be collected from patients with pancreatic ductal adenocarcinoma during routine surgical resection, image-guided percutaneous biopsy, or endoscopic ultrasound-guided fine-needle biopsy (EUS-FNB, 19G or 22G needle recommended), with surgical specimens requiring a volume \>0.5 cm³ and biopsy specimens requiring ≥2 cores of at least 1 cm in length, all having a tumor cell content and viability \>50% with minimal necrosis, fibrosis, fat, and blood contamination (each ≤50%); immediately after collection, samples will be immersed in tissue preservation solution, maintained at 4-8°C, transported to the laboratory within 12 hours, and processed within 24 hours.

Central Study Contacts

Jiabin Jin, MD

CONTACT

0086-18917762352; jjb11501@rjh.com.cn

Zhaohong Wen, PhD

CONTACT

0086-13522210548; wenzhaohong@daxiangbio.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 18 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2026
• First Posted: July 6, 2026
• Study Start: February 20, 2026
• Primary Completion (Estimated): February 20, 2028
• Study Completion (Estimated): February 20, 2028
• Last Updated: July 6, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)