An Exosomal miRNA-based Liquid Biopsy for ICC Detection

NCT07225452 | Completed

• 1 other identifier: 23228/LUMIC
• Study Type: observational
• Target: 535
• Locations: 1 country
• Sites: 1

Brief Summary

Intrahepatic cholangiocarcinoma (ICC) is a malignant liver tumor with poor prognosis and limited curative treatment options. Early and accurate detection remains an unmet clinical need. The LUMIC study aims to develop a non-invasive liquid biopsy platform based on both exosomal microRNAs (exo-miRNAs) to detect intrahepatic cholangiocarcinoma with high sensitivity and specificity.

Conditions

Intrahepatic Cholangiocarcinoma (Icc)

Keywords

ICC; Exosome; microRNA; miRNA; liquid biopsy; liver cancer

Outcome Measures

Primary Outcomes (1)

• Sensitivity

  The proportion of true positive ICC cases correctly identified by the LUMIC assay.

  Through study completion (~1 year)

Secondary Outcomes (2)

• Specificity

  Through study completion (~1 year)

• Diagnostic accuracy (AUC)

  Through study completion (~1 year)

Study Arms (6)

Intrahepatic Cholangiocarcinoma (Discovery, Small RNA-seq)

Serum and plasma samples from patients with histologically confirmed ICC will be analyzed using small RNA sequencing to identify circulating miRNAs specifically upregulated in ICC. These miRNAs will serve as candidates for downstream validation.

Diagnostic Test: Small RNA sequencing

Non-disease Control (Discovery, Small RNA-seq)

Serum and plasma samples from individuals without malignant or inflammatory liver diseases (benign or healthy controls) will be analyzed in parallel by small RNA sequencing to identify miRNAs differentially expressed between ICC and non-disease controls.

Diagnostic Test: Small RNA sequencing

Intrahepatic Cholangiocarcinoma (Training)

Patients with histologically confirmed ICC whose pre-treatment serum or plasma samples will be used to construct and optimize the exo-miRNA diagnostic panel based on discovery-phase candidates.

Diagnostic Test: LUMIC assay

Non-disease Control (Training)

Individuals without malignant or inflammatory liver diseases (benign or healthy controls) whose serum/plasma samples will serve as controls to establish baseline miRNA expression and diagnostic thresholds.

Diagnostic Test: LUMIC assay

Intrahepatic Cholangiocarcinoma (Validation)

Independent ICC cohort used for external validation of the LUMIC assay to confirm diagnostic performance and reproducibility.

Diagnostic Test: LUMIC assay

Non-disease Control (Validation)

Individuals without malignant or inflammatory liver diseases (benign or healthy controls) whose serum/plasma samples will be used for validation of specificity and model robustness.

Diagnostic Test: LUMIC assay

Interventions

Small RNA sequencing DIAGNOSTIC_TEST

Small RNA sequencing of serum/plasma RNA to identify ICC-specific upregulated miRNAs

Intrahepatic Cholangiocarcinoma (Discovery, Small RNA-seq); Non-disease Control (Discovery, Small RNA-seq)

LUMIC assay DIAGNOSTIC_TEST

RT-qPCR validation of selected miRNAs

Intrahepatic Cholangiocarcinoma (Training); Intrahepatic Cholangiocarcinoma (Validation); Non-disease Control (Training); Non-disease Control (Validation)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals diagnosed with intrahepatic cholangiocarcinoma and control participants (non-cancer or benign biliary disease) with available pre-treatment plasma samples.

You may qualify if:

• Age ≥ 18 years
• Histologically confirmed intrahepatic cholangiocarcinoma
• Availability of pre-treatment plasma sample
• Informed consent provided

You may not qualify if:

• Extrahepatic cholangiocarcinoma
• History of other malignancy within 5 years
• Active infection, autoimmune disease, or pregnancy
• Inadequate clinical data or poor sample quality

Sponsors & Collaborators

• City of Hope Medical Center: lead

Study Sites (1)

City of Hope Medical Center

Duarte, California, 91016, United States

Location

Related Publications (12)

• Li J, Bao H, Huang Z, Liang Z, Lin N, Ni C, Xu Y. Non-Coding RNA in Cholangiocarcinoma: An Update. Front Biosci (Landmark Ed). 2023 Aug 18;28(8):173. doi: 10.31083/j.fbl2808173.

  PMID: 37664914 BACKGROUND

• Sato K, Glaser S, Alvaro D, Meng F, Francis H, Alpini G. Cholangiocarcinoma: novel therapeutic targets. Expert Opin Ther Targets. 2020 Apr;24(4):345-357. doi: 10.1080/14728222.2020.1733528. Epub 2020 Feb 26.

  PMID: 32077341 BACKGROUND

• Luo C, Xin H, Zhou Z, Hu Z, Sun R, Yao N, Sun Q, Borjigin U, Wu X, Fan J, Huang X, Zhou S, Zhou J. Tumor-derived exosomes induce immunosuppressive macrophages to foster intrahepatic cholangiocarcinoma progression. Hepatology. 2022 Oct;76(4):982-999. doi: 10.1002/hep.32387. Epub 2022 Feb 28.

  PMID: 35106794 BACKGROUND

• Li Z, Shen J, Chan MT, Wu WK. The role of microRNAs in intrahepatic cholangiocarcinoma. J Cell Mol Med. 2017 Jan;21(1):177-184. doi: 10.1111/jcmm.12951. Epub 2016 Sep 13.

  PMID: 27619971 BACKGROUND

• Wada Y, Shimada M, Morine Y, Ikemoto T, Saito Y, Baba H, Mori M, Goel A. A blood-based noninvasive miRNA signature for predicting survival outcomes in patients with intrahepatic cholangiocarcinoma. Br J Cancer. 2022 May;126(8):1196-1204. doi: 10.1038/s41416-022-01710-z. Epub 2022 Jan 25.

  PMID: 35079106 BACKGROUND

• Liu L, Shi Y, Zhang P, Zhang X. Integration analysis of miRNA-mRNA expression exploring their potential roles in intrahepatic cholangiocarcinoma. Sci Rep. 2023 May 24;13(1):8362. doi: 10.1038/s41598-023-35288-0.

  PMID: 37225858 BACKGROUND

• European Association for the Study of the Liver. EASL-ILCA Clinical Practice Guidelines on the management of intrahepatic cholangiocarcinoma. J Hepatol. 2023 Jul;79(1):181-208. doi: 10.1016/j.jhep.2023.03.010. Epub 2023 Apr 20.

  PMID: 37084797 BACKGROUND

• Bridgewater J, Galle PR, Khan SA, Llovet JM, Park JW, Patel T, Pawlik TM, Gores GJ. Guidelines for the diagnosis and management of intrahepatic cholangiocarcinoma. J Hepatol. 2014 Jun;60(6):1268-89. doi: 10.1016/j.jhep.2014.01.021. Epub 2014 Mar 27. No abstract available.

  PMID: 24681130 BACKGROUND

• Moris D, Palta M, Kim C, Allen PJ, Morse MA, Lidsky ME. Advances in the treatment of intrahepatic cholangiocarcinoma: An overview of the current and future therapeutic landscape for clinicians. CA Cancer J Clin. 2023 Mar;73(2):198-222. doi: 10.3322/caac.21759. Epub 2022 Oct 19.

  PMID: 36260350 BACKGROUND

• Tovar-Camargo OA, Toden S, Goel A. Exosomal microRNA Biomarkers: Emerging Frontiers in Colorectal and Other Human Cancers. Expert Rev Mol Diagn. 2016;16(5):553-67. doi: 10.1586/14737159.2016.1156535. Epub 2016 Mar 16.

  PMID: 26892862 BACKGROUND

• Sui S, Xu C, Kanda M, Okugawa Y, Toiyama Y, Park JO, Hur H, Kim SC, Taketomi A, Kodera Y, Cheng X, Li M, Goel A. Exosomal Liquid Biopsy for the Early Detection of Gastric Cancer: The DESTINEX Multicenter Study. JAMA Surg. 2025 Sep 1;160(9):973-982. doi: 10.1001/jamasurg.2025.2493.

  PMID: 40737022 BACKGROUND

• Nakamura K, Zhu Z, Roy S, Jun E, Han H, Munoz RM, Nishiwada S, Sharma G, Cridebring D, Zenhausern F, Kim S, Roe DJ, Darabi S, Han IW, Evans DB, Yamada S, Demeure MJ, Becerra C, Celinski SA, Borazanci E, Tsai S, Kodera Y, Park JO, Bolton JS, Wang X, Kim SC, Von Hoff D, Goel A. An Exosome-based Transcriptomic Signature for Noninvasive, Early Detection of Patients With Pancreatic Ductal Adenocarcinoma: A Multicenter Cohort Study. Gastroenterology. 2022 Nov;163(5):1252-1266.e2. doi: 10.1053/j.gastro.2022.06.090. Epub 2022 Jul 16.

  PMID: 35850192 BACKGROUND

MeSH Terms

Conditions

Cholangiocarcinoma; Cirrhosis, Familial, with Pulmonary Hypertension; Liver Neoplasms

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Study Officials

• Ajay Goel, PhD

  City of Hope Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: November 4, 2025
• First Posted: November 6, 2025
• Study Start: June 21, 2024
• Primary Completion: January 5, 2026
• Study Completion: February 5, 2026
• Last Updated: March 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)