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Clinical Trials/NCT03959800
NCT03959800
Recruiting
Not Applicable

Genetic and Molecular Basis of Pediatric Liver Cancer

University of Pittsburgh1 site in 1 country1,600 target enrollmentJune 22, 2015

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Childhood Liver Cancer
Sponsor
University of Pittsburgh
Enrollment
1600
Locations
1
Primary Endpoint
Gene sequencing
Status
Recruiting
Last Updated
7 months ago

Overview

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.

Registry
clinicaltrials.gov
Start Date
June 22, 2015
End Date
June 30, 2029
Last Updated
7 months ago
Study Type
Observational
Sex
All

Investigators

Responsible Party
Principal Investigator
Principal Investigator

Rakesh Sindhi

Professor of Surgery

University of Pittsburgh

Eligibility Criteria

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.

Outcomes

Primary Outcomes

Gene sequencing

Time Frame: Recurrence free survival at 2 years

DNA sequence variants

Status of genome-wide chromatin accessibility

Time Frame: Duration of active chemotherapy to two years after surgical treatment

chromatin accessibility

Gene expression analysis

Time Frame: Recurrence free survival at 2 years

Differentially expressed genes

Tumor infiltrating cells which express immune checkpoints

Time Frame: Duration of active chemotherapy to two years after surgical treatment

differentially enriched immune cells

Epigenetic change

Time Frame: Duration of active chemotherapy to two years after surgical treatment

Differential methylation

Secondary Outcomes

  • Response to chemotherapy(Duration of active chemotherapy to two years after surgical treatment)

Study Sites (1)

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