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Molecular Basis of Pediatric Liver Cancer

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ClinicalTrials.gov Identifier: NCT03959800
Recruitment Status : Recruiting
First Posted : May 22, 2019
Last Update Posted : May 22, 2019
Sponsor:
Information provided by (Responsible Party):
Rakesh Sindhi, 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.

Condition or disease
Childhood Liver Cancer Liver Malignant Tumors Embryonal Sarcoma of Liver (Disorder) Hepatoblastoma Hepatocellular Carcinoma Rhabdoid Tumor of Liver

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.


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Study Type : Observational
Estimated Enrollment : 1600 participants
Observational Model: Other
Time Perspective: Other
Official Title: Genetic and Molecular Basis of Pediatric Liver Cancer
Actual Study Start Date : June 22, 2015
Estimated Primary Completion Date : June 30, 2028
Estimated Study Completion Date : June 30, 2029





Primary Outcome Measures :
  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 Outcome Measures :
  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


Biospecimen Retention:   Samples With DNA
Samples with DNA: blood, saliva or liver tissue.


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Ages Eligible for Study:   up to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Living or deceased individuals who were diagnosed with cancer of the liver during childhood (age <21 years) and their biological parents and siblings.
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03959800


Contacts
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Contact: Alexandra Kepler, MPH (412) 692-6692 alexandra.kepler@upmc.edu
Contact: Alexis Rzempoluch, MS (412) 692-5201 alexis.rzempoluch@chp.edu

Locations
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United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Alexandra Kepler, MPH    412-692-6692    alexandra.kepler@upmc.edu   
Contact: Alexis Rzempoluch, MS    412-692-5201    alexis.rzempoluch@chp.edu   
Principal Investigator: Rakesh Sindhi, MD         
Sponsors and Collaborators
University of Pittsburgh

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Responsible Party: Rakesh Sindhi, Professor of Surgery, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT03959800     History of Changes
Other Study ID Numbers: PRO14120080
First Posted: May 22, 2019    Key Record Dates
Last Update Posted: May 22, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Rakesh Sindhi, University of Pittsburgh:
Hepatoblastoma
Hepatocellular Carcinoma
Pediatric Liver Cancer

Additional relevant MeSH terms:
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Carcinoma, Hepatocellular
Liver Neoplasms
Neoplasms
Rhabdoid Tumor
Hepatoblastoma
Adenocarcinoma
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases
Neoplasms, Complex and Mixed