Molecular Profiling for Risk Stratification in Appendiceal Cancer

NCT07587268 | Recruiting

• 1 other identifier: 23228_PACE
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief Summary

This study investigates integrated epigenetic and epitranscriptomic features of appendiceal cancer using archived tumor tissue specimens from the same patient cohort. The study includes DNA methylation profiling and m6A epitranscriptomic profiling to define molecular subtypes, evaluate associations with clinicopathologic features, and develop molecular risk scores for prognostic stratification. The primary goal is to determine whether DNA methylation- and m6A-based molecular features can complement conventional histopathologic grading and improve risk stratification.

Conditions

Appendiceal Cancer; Appendiceal Adenocarcinoma

Keywords

Appendiceal cancer; Appendiceal adenocarcinoma; DNA methylation; m6A modification; Multi-omics; MeRIP-seq; Histologic grade;; Epigenomics; Epitranscriptomics; Molecular subtype; Risk stratification; Prognostic biomarker

Outcome Measures

Primary Outcomes (2)

• Overall Survival

  Overall survival will be evaluated in relation to molecular profiling results and available clinicopathologic characteristics. Survival analyses may include Kaplan-Meier analysis and Cox proportional hazards models

  Through study completion, an average of 1 year

• Progression-Free Survival

  Progression-free survival will be evaluated in relation to molecular profiling results and available clinicopathologic characteristics. Time-to-event analyses may be performed using standard survival analysis methods.

  Through study completion, an average of 1 year

Study Arms (2)

Appendiceal Cancer Cohort

Patients with histologically confirmed appendiceal cancer whose archived tumor tissue specimens and clinicopathologic data are available for integrated DNA methylation and m6A epitranscriptomic profiling.

Diagnostic Test: DNA Methylation Profiling; Diagnostic Test: m6A Epitranscriptomic Profiling

Benign Appendix Reference Cohort

Individuals with benign or normal appendix tissue specimens used as non-malignant reference samples for comparison of tumor-associated DNA methylation and m6A epitranscriptomic features.

Diagnostic Test: DNA Methylation Profiling; Diagnostic Test: m6A Epitranscriptomic Profiling

Interventions

DNA Methylation Profiling DIAGNOSTIC_TEST

Archived tissue specimens undergo DNA methylation profiling to characterize tumor-associated methylation alterations and identify methylation-based molecular features associated with clinicopathologic variables and survival outcomes. The profiling is performed for research purposes only and does not assign treatment.

Appendiceal Cancer Cohort; Benign Appendix Reference Cohort

m6A Epitranscriptomic Profiling DIAGNOSTIC_TEST

Archived tissue specimens undergo m6A methylated RNA immunoprecipitation sequencing with matched input RNA sequencing to quantify transcriptome-wide m6A enrichment. The resulting molecular data are used for subtype discovery, m6A score construction, reduced-panel development, and association with clinicopathologic and survival outcomes.

Appendiceal Cancer Cohort; Benign Appendix Reference Cohort

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

The study population includes adult patients with appendiceal cancer whose archived tumor specimens and associated clinicopathologic data are available for retrospective molecular profiling. The same patient cohort is used for DNA methylation and m6A epitranscriptomic analyses where sufficient biospecimen material is available. Benign or normal appendix tissue specimens may be included as non-malignant reference controls.

You may qualify if:

• Patients with histologically confirmed appendiceal adenocarcinoma or appendiceal cancer.
• Availability of archived tumor tissue suitable for molecular profiling.
• Availability of tissue for DNA methylation profiling, m6A epitranscriptomic profiling, or both.
• Availability of relevant clinicopathologic data.
• Availability of survival or follow-up information when applicable.
• Age 18 years or older at diagnosis or tissue collection.

You may not qualify if:

• Insufficient tissue quantity or quality for molecular profiling.
• Inadequate DNA or RNA quality for sequencing or molecular assay preparation.
• Missing essential clinicopathologic information required for analysis.
• Non-appendiceal primary tumor or metastatic tumor to the appendix from another primary site.
• Patients who do not meet institutional review board or consent requirements, if applicable.

Sponsors & Collaborators

• City of Hope Medical Center: lead

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91016, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Archived formalin-fixed paraffin-embedded appendiceal cancer tissue specimens and, where available, benign or normal appendix tissue specimens are used for DNA methylation profiling and m6A epitranscriptomic profiling. Matched clinicopathologic and survival data are collected from medical records.

MeSH Terms

Conditions

Appendiceal Neoplasms; Adenocarcinoma, Mucinous

Condition Hierarchy (Ancestors)

Cecal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Cecal Diseases; Intestinal Diseases; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Cystic, Mucinous, and Serous

Central Study Contacts

Ajay Goel

CONTACT

6262184673; ajgoel@coh.org

Study Design

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

Study Record Dates

• First Submitted: May 4, 2026
• First Posted: May 14, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: May 14, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)