A Personalized Surveillance and Intervention Protocol for Duodenal and Gastric Polyposis in Patients With Familial Adenomatous Polyposis

The purpose of this study is to determine the efficacy and safety of a personalized surveillance and intervention protocol for duodenal and gastric polyposis in patients with familial adenomatous polyposis (FAP)

Study Overview

• Status: Recruiting
• Conditions: Familial Adenomatous Polyposis
• Intervention / Treatment: Procedure: Personalized surveillance and intervention protocol

Detailed Description

Patients with FAP are not only at risk of developing colorectal adenomas but also at high risk of developing duodenal adenomas. In 30% to 92% of FAP patients duodenal adenomas are detected, with a lifetime risk approaching 100%. Of these duodenal adenomas, only a small proportion develops into duodenal cancer, with a prevalence of approximately 5-10% in FAP patients.

Endoscopic surveillance is nowadays the standard of care to prevent FAP patients from developing duodenal cancer. The severity of duodenal polyposis is assessed using the Spigelman classification system. This classification is based on the number, size, histology, and grade of dysplasia of the duodenal adenomas, resulting in a score varying from 0-IV, guiding surveillance intervals and treatment.

Concerns are rising on the accuracy of the Spigelman score as predictor for duodenal cancer, especially for ampullary cancer. Over the past years, multiple studies demonstrated limitations of this staging system including the fact that this classification does not adequately predict duodenal/ampullary cancer and does not guide endoscopic or surgical interventions. A clear endoscopic intervention protocol is needed, not only to prevent the development of cancer but also to prevent the need for duodenal surgery, since these surgical procedures are associated with high complication and mortality rates.

With this study, the investigators aim to evaluate a personalized surveillance and intervention protocol for the duodenum and stomach with the goal to prevent the development of advanced neoplasia (AN) by endoscopically removing lesions before they progress to AN.

• Study Type: Observational
• Enrollment (Anticipated): 1000

Contacts and Locations

Study Locations

• Netherlands
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1105AZ
      • Recruiting
      • Academic Medical Centre
      • Contact: Evelien Dekker, MD, PhD; Phone Number: 0031205661260; Email: e.dekker@amc.uva.nl
      • Sub-Investigator: Arthur Aelvoet, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Patients with FAP treated at one of the participating centers will be assessed for eligibility.

Description

Inclusion Criteria:

• Diagnosis of FAP, at least one of following: genetic diagnosis (proven APC germline mutation) and/or clinical diagnosis (>100 colorectal adenomas in combination with a positive family history of FAP)
• Age 18 years or older

Exclusion Criteria:

• Endoscopic removal of all polyps with an indication for removal not possible/feasible
• Gastric or duodenal cancer at baseline endoscopy
• Need for surgery

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Personalized surveillance and intervention protocol	Procedure: Personalized surveillance and intervention protocol; This study uses one arm. Participants will undergo endoscopic surveillance with intervals between 3-6 months and 5 years, depending on severity of polyposis and performed endoscopic interventions.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Advanced neoplasia	Incidence of advanced neoplasia defined as adenomas ≥15mm, high grade dysplasia (HGD) and/or duodenal/ampullary cancer	Up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Recurrences after different endoscopic intervention techniques	Incidence of recurrences after endoscopic interventions after en bloc/piecemeal resection and different techniques such as cold snare polypectomy or endoscopic mucosal resection with or without lifting	Analysis at 2 years and 5 years
Feasibility of endoscopic interventions	Incidence of lesions not amenable to endoscopic removal	Analysis at 2 years and 5 years
Accuracy optical diagnosis	The ability of endoscopists to optically diagnose duodenal and gastric lesions. Sensitivity and specificity for optically diagnose high-grade dysplasia in the stomach and duodenum.	Analysis at 2 years and 5 years
Complications	Incidence of endoscopy related complications	Analysis at 2 years and 5 years
Surveillance burden	Surveillance burden (number of endoscopies for each patient)	Up to 5 years
Surgery	Incidence of surgical interventions	Up to 5 years

Collaborators and Investigators

• Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Collaborators: IRCCS Azienda Ospedaliero-Universitaria di Bologna; Hospital Clinic of Barcelona; St Mark's Hospital Foundation; Hôpital Edouard Herriot; The Netherlands Cancer Institute; Leiden University Medical Center; Maria Sklodowska-Curie National Research Institute of Oncology; Hospital General Universitario de Alicante; University Medical Center Mainz

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 24, 2020
• Primary Completion (ANTICIPATED): November 1, 2025
• Study Completion (ANTICIPATED): November 1, 2025

Study Registration Dates

• First Submitted: November 24, 2020
• First Submitted That Met QC Criteria: December 16, 2020
• First Posted (ACTUAL): December 21, 2020

Study Record Updates

• Last Update Posted (ACTUAL): December 21, 2020
• Last Update Submitted That Met QC Criteria: December 16, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: Gastrointestinal neoplasia; Personalized care; Endoscopic surveillance; Endoscopic interventions
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Gastrointestinal Neoplasms; Digestive System Neoplasms; Gastrointestinal Diseases; Genetic Diseases, Inborn; Pharyngeal Neoplasms; Otorhinolaryngologic Neoplasms; Head and Neck Neoplasms; Nasopharyngeal Diseases; Pharyngeal Diseases; Stomatognathic Diseases; Otorhinolaryngologic Diseases; Colonic Diseases; Intestinal Diseases; Intestinal Neoplasms; Rectal Diseases; Neoplastic Syndromes, Hereditary; Adenomatous Polyps; Adenoma; Intestinal Polyposis; Nasopharyngeal Neoplasms; Colorectal Neoplasms; Adenomatous Polyposis Coli
• Other Study ID Numbers: W20_181

Drug and device information, study documents

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