Childhood Liver CancerNCT03959800
Molecular Basis of Pediatric Liver Cancer
An observational study of Childhood Liver Cancer and Liver Malignant Tumors, run by University of Pittsburgh at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 99 years
- Conditions the study is for
- Childhood Liver Cancer, Liver Malignant Tumors, Embryonal Sarcoma of Liver (Disorder)
- How many people
- 1,600 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jun 2015to 30 Jun 2035
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Prior or current treatment for a childhood liver tumor, malignant or benign, at age <21 years.
- Biological parents and siblings of eligible children.
Who cannot join
- No prior or current treatment for a childhood liver tumor.
- Non-biological parents, legal guardians, or non-biological siblings of eligible children.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Morgan L Paul, BSN, 412-692-8472, Morgan.Paul2@upmc.edu
Daniel Pieratt, MPA, 4126926692, pierattdw@upmc.edu
About this study
Official title: Genetic and 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.
Read the 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.
- 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
- Medical subject headings
- Carcinoma, Hepatocellular, Hepatoblastoma
What is being measured
The primary outcome is the main question the study is designed to answer.
Gene sequencing
DNA sequence variants
Measured: Recurrence free survival at 2 years
Gene expression analysis
Differentially expressed genes
Measured: Recurrence free survival at 2 years
Status of genome-wide chromatin accessibility
chromatin accessibility
Measured: Duration of active chemotherapy to two years after surgical treatment
Epigenetic change
Differential methylation
Measured: Duration of active chemotherapy to two years after surgical treatment
Tumor infiltrating cells which express immune checkpoints
differentially enriched immune cells
Measured: Duration of active chemotherapy to two years after surgical treatment
Secondary outcomes (2)
Response to chemotherapy
Survival
Measured: Duration of active chemotherapy to two years after surgical treatment
Response to chemotherapy
Relapse
Measured: Duration of active chemotherapy to two years after surgical treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2019
- Study start22 Jun 2015
- Primary completion30 Jun 2030 (estimated)
- Study completion30 Jun 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Liver CancerStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 1,600 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rakesh Sindhi, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03959800
- Sponsor's protocol ID
- STUDY20050018
- Status last verified
- Aug 2026
- First submitted
- 15 May 2019
- Last updated
- 10 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Genetic and Molecular Basis of Pediatric Liver Cancer
- ClinicalTrials.gov ID
- NCT03959800
- Acronym / sponsor ID
- STUDY20050018
- 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
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,600 participants (estimated)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Study start
- 22 Jun 2015 (actual)
- Primary completion
- 30 Jun 2030 (estimated)
- Study completion
- 30 Jun 2035 (estimated)
- First posted
- 22 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Ages
- 0 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- Prior or current treatment for a childhood liver tumor, malignant or benign, at age <21 years.
- Biological parents and siblings of eligible children.
Key exclusion criteria (2 listed)
- No prior or current treatment for a childhood liver tumor.
- Non-biological parents, legal guardians, or non-biological siblings of eligible children.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Gene sequencing
Time frame: Recurrence free survival at 2 years
DNA sequence variants
Gene expression analysis
Time frame: Recurrence free survival at 2 years
Differentially expressed genes
Status of genome-wide chromatin accessibility
Time frame: Duration of active chemotherapy to two years after surgical treatment
chromatin accessibility
Epigenetic change
Time frame: Duration of active chemotherapy to two years after surgical treatment
Differential methylation
Tumor infiltrating cells which express immune checkpoints
Time frame: Duration of active chemotherapy to two years after surgical treatment
differentially enriched immune cells
2 secondary outcomes
Response to chemotherapy
Time frame: Duration of active chemotherapy to two years after surgical treatment
Response to chemotherapy
Time frame: Duration of active chemotherapy to two years after surgical treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Childhood Liver Cancer and Liver Malignant Tumors, run by University of Pittsburgh at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Morgan Paul, BSN ((412) 692-8472, Morgan.Paul2@upmc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Gene sequencing (time frame: Recurrence free survival at 2 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2026.