A Cell-free and Exosomal miRNA-based Liquid Biopsy for ICC Detection (LUMIC)

City of Hope

Status: Enrolling

Conditions: Intrahepatic Cholangiocarcinoma (Icc)

Treatments: Diagnostic Test: LUMIC assay; Diagnostic Test: Small RNA sequencing

Study type: Observational

Funder types: Other

Identifiers

NCT07225452

23228/LUMIC

Details and patient eligibility

About

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 cell-free and exosomal microRNAs (cf- and exo-miRNAs) to detect intrahepatic cholangiocarcinoma with high sensitivity and specificity.

Full description

Intrahepatic cholangiocarcinoma (ICC) is the second most common primary liver malignancy after hepatocellular carcinoma, accounting for approximately 10-15% of all primary liver cancers. Despite improvements in surgical techniques and imaging modalities, ICC is often diagnosed at advanced stages, resulting in dismal outcomes with a 5-year overall survival rate of 25-30%.

Traditional imaging approaches such as CT and MRI have limited sensitivity for detecting early or small ICC lesions. Blood-based biomarkers, including CA19-9, also lack adequate specificity.

Recent advances in liquid biopsy have demonstrated that circulating cell-free and exosomal microRNAs (cf- and exo-miRNAs) can serve as promising, minimally invasive biomarkers reflecting tumor biology and microenvironmental changes.

The LUMIC study (Liquid biopsy Using cell-free and exosomal miRNA for Intrahepatic Cholangiocarcinoma detection) aims to identify and validate miRNA signatures capable of distinguishing ICC from benign biliary or non-cancerous liver conditions.

Blood samples are collected before treatment, and cf-/exo-miRNA expression profiles are analyzed using RT-qPCR and bioinformatic pipelines. Diagnostic performance (AUC, sensitivity, specificity) will be evaluated through training and validation cohorts.

This study provides a foundation for integrating liquid biopsy-based diagnostics into ICC clinical workflows to enable earlier detection and improved treatment stratification.

Enrollment: 500 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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

Trial design

500 participants in 6 patient groups

Intrahepatic Cholangiocarcinoma (Discovery, Small RNA-seq)

Description:

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.

Treatment:

Diagnostic Test: Small RNA sequencing

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

Description:

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.

Treatment:

Diagnostic Test: Small RNA sequencing

Intrahepatic Cholangiocarcinoma (Training)

Description:

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

Treatment:

Diagnostic Test: LUMIC assay

Non-disease Control (Training)

Description:

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.

Treatment:

Diagnostic Test: LUMIC assay

Intrahepatic Cholangiocarcinoma (Validation)

Description:

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

Treatment:

Diagnostic Test: LUMIC assay

Non-disease Control (Validation)

Description:

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.

Treatment:

Diagnostic Test: LUMIC assay