IPEX Study: Pancreatic Exocrine Insufficiency as a Functional Marker of Disease Progression in Patients With IPMN

NCT07535125 | Not Yet Recruiting

• 1 other identifier: IPEX
• Study Type: observational
• Target: 600
• Locations: 0 countries
• Sites: N/A

Brief Summary

Intraductal Papillary Mucinous Neoplasms (IPMN) are pancreatic cystic neoplasms managed through imaging-based surveillance focused on oncologic risk. However, IPMN pathophysiology includes ductal obstruction by mucin, chronic ductal hypertension, and progressive parenchymal atrophy, mechanisms that may lead to pancreatic exocrine insufficiency (PEI). PEI is a maldigestion syndrome typically associated with chronic pancreatitis, pancreatic cancer, and pancreatic surgery, but it has never been systematically investigated in patients with IPMN under surveillance. The IPEX study is a multicenter prospective observational cohort study designed to evaluate whether PEI is prevalent in IPMN patients and whether it correlates with morphologic disease progression. The study also evaluates the potential role of PEI as a functional marker complementary to imaging criteria in IPMN surveillance.

Conditions

Intraductal Papillary Mucinous Neoplasms; Pancreatic Exocrine Insufficiency

Keywords

Functional Marker; pancreatic exocrine insufficiency; Intraductal Papillary Mucinous Neoplasms

Outcome Measures

Primary Outcomes (1)

• Association between clinically relevant PEI and IPMN progression during follow-up

  Clinically relevant PEI defined as: * FE-1 \<100 µg/g OR * FE-1 100-200 µg/g plus ≥1 clinical criterion IPMN progression defined by WF/HRS development, surgical referral, or histologic progression

  Day 1 and after 6 and 12 months

Secondary Outcomes (5)

• Prevalence and incidence of PEI in IPMN patients

  Day 1 and after 6 and 12 months

• Association between imaging parameters (MPD diameter, parenchymal atrophy) and PEI

  Day 1 and after 6 and 12 months

• Time-to-progression stratified by PEI status

  Day 1 and after 6 and 12 months

• Nutritional impact of PEI

  Day 1 and after 6 and 12 months

• Incremental predictive value of PEI beyond imaging criteria

  Day 1 and after 6 and 12 months

Study Arms (1)

IPMN

This study includes a single prospective observational cohort of adult patients (≥18 years) with a diagnosis of intraductal papillary mucinous neoplasm (IPMN)-including branch-duct, main-duct, or mixed-type. Participants are managed according to standard-of-care IPMN surveillance protocols (MRI/MRCP ± EUS), with no mandated interventional treatment. In parallel, patients undergo a structured pancreatology assessment to evaluate pancreatic exocrine function. The exposure of interest is pancreatic exocrine insufficiency (PEI), defined using a composite clinical-biochemical criterion based on fecal elastase-1 (FE-1) levels and standardized clinical and nutritional parameters. No experimental intervention is administered; initiation of pancreatic enzyme replacement therapy (PERT), when clinically indicated, is recorded but not protocol-driven.

Eligibility Criteria

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

Study Population

Patients undergoing routine surveillance for IPMN at participating centers.

You may qualify if:

• Age ≥18 years
• Diagnosis of BD-IPMN, MD-IPMN, or mixed IPMN
• Under active imaging surveillance
• Signed informed consent

You may not qualify if:

• Prior pancreatic surgery
• Chronic pancreatitis
• Known pancreatic ductal adenocarcinoma at baseline
• Other established causes of PEI unrelated to IPMN

Sponsors & Collaborators

• A.O.U. Città della Salute e della Scienza: lead

Related Publications (3)

• Ohtsuka T, Fernandez-Del Castillo C, Furukawa T, Hijioka S, Jang JY, Lennon AM, Miyasaka Y, Ohno E, Salvia R, Wolfgang CL, Wood LD. International evidence-based Kyoto guidelines for the management of intraductal papillary mucinous neoplasm of the pancreas. Pancreatology. 2024 Mar;24(2):255-270. doi: 10.1016/j.pan.2023.12.009. Epub 2023 Dec 28.

  PMID: 38182527 RESULT

• Tanaka M, Fernandez-del Castillo C, Adsay V, Chari S, Falconi M, Jang JY, Kimura W, Levy P, Pitman MB, Schmidt CM, Shimizu M, Wolfgang CL, Yamaguchi K, Yamao K; International Association of Pancreatology. International consensus guidelines 2012 for the management of IPMN and MCN of the pancreas. Pancreatology. 2012 May-Jun;12(3):183-97. doi: 10.1016/j.pan.2012.04.004. Epub 2012 Apr 16.

  PMID: 22687371 RESULT

• Dominguez-Munoz JE, Vujasinovic M, de la Iglesia D, Cahen D, Capurso G, Gubergrits N, Hegyi P, Hungin P, Ockenga J, Paiella S, Perkhofer L, Rebours V, Rosendahl J, Salvia R, Scheers I, Szentesi A, Bonovas S, Piovani D, Lohr JM; European PEI Multidisciplinary Group. European guidelines for the diagnosis and treatment of pancreatic exocrine insufficiency: UEG, EPC, EDS, ESPEN, ESPGHAN, ESDO, and ESPCG evidence-based recommendations. United European Gastroenterol J. 2025 Feb;13(1):125-172. doi: 10.1002/ueg2.12674. Epub 2024 Dec 5.

  PMID: 39639485 RESULT

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases

Study Officials

• Giacomo Deiro, MD

  A.O.U. Città della Salute e della Scienza

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Giacomo Deiro, MD

CONTACT

+393489818794; giacomo.deiro@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2026
• First Posted: April 16, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be available beginning 6-12 months after publication of the primary results and for up to 5 years thereafter.
• Access Criteria: Data sharing will require a formal data-sharing agreement and compliance with applicable data protection laws (e.g., GDPR).