Clinical trial NCT03959800: Molecular Basis of Pediatric Liver Cancer
Official title: Genetic and Molecular Basis of Pediatric Liver Cancer
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Childhood Liver Cancer
Liver Malignant Tumors
Embryonal Sarcoma of Liver (Disorder)
Hepatoblastoma
Hepatocellular Carcinoma
Rhabdoid Tumor of Liver
Keywords: Hepatoblastoma
Hepatocellular Carcinoma
Pediatric Liver Cancer
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
First posted: 2019-05-22
Enrollment: 1600
Sex: ALL
Minimum age (years): 0
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Living or deceased individuals who were diagnosed with a liver tumor, malignant or benign, during childhood (age \<21 years) and their biological parents and siblings.

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.

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.

Primary outcomes:
measure: Gene sequencing
timeFrame: Recurrence free survival at 2 years
measure: Gene expression analysis
timeFrame: Recurrence free survival at 2 years
measure: Status of genome-wide chromatin accessibility
timeFrame: Duration of active chemotherapy to two years after surgical treatment
measure: Epigenetic change
timeFrame: Duration of active chemotherapy to two years after surgical treatment
measure: Tumor infiltrating cells which express immune checkpoints
timeFrame: Duration of active chemotherapy to two years after surgical treatment
Secondary outcomes:
measure: Response to chemotherapy
timeFrame: Duration of active chemotherapy to two years after surgical treatment
measure: Response to chemotherapy
timeFrame: Duration of active chemotherapy to two years after surgical treatment
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
Study officials:
References: