Molecular Basis of Pediatric Liver Cancer

Recruiting

• Conditions: Liver Malignant Tumors; Hepatoblastoma; Embryonal Sarcoma of Liver (Disorder); Hepatocellular Carcinoma; Rhabdoid Tumor of Liver; Childhood Liver Cancer

• Registration Number: NCT03959800
• Lead Sponsor: University of Pittsburgh

Brief Summary

The purpose of this retrospective and prospective project is to understand the molecular and genetic basis of liver cancer of childhood. Understanding the molecular and genetic bases of liver cancers can offer a better classification based on tumor biology, mechanisms and predisposition.

Detailed Description

Pediatric liver cancers are rare, affecting at times no more than 1 in one million population. Understanding the molecular basis of these cancers is important in order to develop more accurate diagnoses and more effective treatments. Current classifications of these cancers are based on how these cancers look on diagnostic studies such as radiologic imaging or under the microscope. Such a classification system does not explain why a particular cancer has a different outcome from what is considered "usual" for that particular cancer. Nor does such a classification system explain why two different classes of cancers behave the same way. Understanding the genetic bases of liver cancers can offer a better classification based on tumor biology, mechanisms and predisposition.

To achieve these goals, large numbers of such cancer patients or affected tissue must be collected. This is not possible in any single institution, or any single country. The current project will collect biological samples such as residual tumor tissue, saliva, or blood from affected patients and their biological parents and families, along with clinical information about the cancer. These biological samples will be used to study the genes and how these genes work in tumor tissue and in non-tumor tissue. The results of this study will permit childhood liver cancers to be categorized on the basis of common defects in genes and their function.

Study Timeline

• Study Start: 2015-06-22
• Study First Submitted: 2019-05-15
• Study First Submitted QC: 2019-05-21
• Study First Posted: 2019-05-22
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-04
• Last Update Posted: 2025-02-07
• Primary Completion: 2028-06-30
• Study Completion: 2029-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1600

Inclusion Criteria

• Prior or current treatment for a childhood liver tumor, malignant or benign, at age <21 years.
• Biological parents and siblings of eligible children.

Exclusion Criteria

• No prior or current treatment for a childhood liver tumor.
• Non-biological parents, legal guardians, or non-biological siblings of eligible children.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Gene sequencing	Recurrence free survival at 2 years	DNA sequence variants
Status of genome-wide chromatin accessibility	Duration of active chemotherapy to two years after surgical treatment	chromatin accessibility
Gene expression analysis	Recurrence free survival at 2 years	Differentially expressed genes
Tumor infiltrating cells which express immune checkpoints	Duration of active chemotherapy to two years after surgical treatment	differentially enriched immune cells
Epigenetic change	Duration of active chemotherapy to two years after surgical treatment	Differential methylation

Secondary Outcome Measures

Name	Time	Method
Response to chemotherapy	Duration of active chemotherapy to two years after surgical treatment	Relapse

Trial Locations

Locations (1)

UPMC Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States