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NCT Number: NCT03959800

Molecular Basis of Pediatric Liver Cancer

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.

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Key information

Age range

0 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location status: Recruiting

Location contact

Daniel Pieratt, MPA

CONTACT

[email protected]

4126926692

Morgan L Paul, BSN

CONTACT

[email protected]

412-692-8472

Rakesh Sindhi, MD

PRINCIPAL_INVESTIGATOR

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Gene sequencing

    Time frame: Recurrence free survival at 2 years

    DNA sequence variants

  2. Gene expression analysis

    Time frame: Recurrence free survival at 2 years

    Differentially expressed genes

  3. Status of genome-wide chromatin accessibility

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

    chromatin accessibility

  4. Epigenetic change

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

    Differential methylation

  5. Tumor infiltrating cells which express immune checkpoints

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

    differentially enriched immune cells

Secondary outcomes

  1. Response to chemotherapy

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

    Survival

  2. Response to chemotherapy

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

    Relapse

Study contacts

Contact information is provided by the study sponsor or research team.

Daniel Pieratt, MPA

CONTACT

[email protected]

(412) 692-6692

Morgan Paul, BSN

CONTACT

[email protected]

(412) 692-8472

Sponsors and collaborators

Lead sponsor

University of Pittsburgh

Other

Registry information

Official study title

Genetic and Molecular Basis of Pediatric Liver Cancer

Important dates

Study start
2015
Primary completion
2028
Study completion
2029
First posted
May 22, 2019
Registry last updated
Sep 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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