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Molecular Basis of Pediatric Liver Cancer
| Condition or disease |
|---|
| Childhood Liver Cancer Liver Malignant Tumors Embryonal Sarcoma of Liver (Disorder) Hepatoblastoma Hepatocellular Carcinoma Rhabdoid Tumor of Liver |
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 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 |
- 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
- Response to chemotherapy [ Time Frame: Duration of active chemotherapy to two years after surgical treatment ]Survival
- Response to chemotherapy [ Time Frame: Duration of active chemotherapy to two years after surgical treatment ]Relapse
Biospecimen Retention: Samples With DNA
| 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 |
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.
| Contact: Alexandra Kepler, MPH | (412) 692-6692 | alexandra.kepler@upmc.edu | |
| Contact: Tara Sherman, MS | (412) 692-5201 | drapertv@upmc.edu |
| 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: Tara Sherman, MS 412-692-5201 drapertv@upmc.edu | |
| Principal Investigator: Rakesh Sindhi, MD | |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date | May 15, 2019 | ||||||||
| First Posted Date | May 22, 2019 | ||||||||
| Last Update Posted Date | June 4, 2021 | ||||||||
| Actual Study Start Date | June 22, 2015 | ||||||||
| Estimated Primary Completion Date | June 30, 2028 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures |
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| Original Primary Outcome Measures | Same as current | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures |
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| Original Secondary Outcome Measures | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title | Molecular Basis of Pediatric Liver Cancer | ||||||||
| Official Title | Genetic and Molecular Basis of Pediatric Liver Cancer | ||||||||
| 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. |
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| Study Type | Observational | ||||||||
| Study Design | Observational Model: Other Time Perspective: Other |
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| Target Follow-Up Duration | Not Provided | ||||||||
| Biospecimen | Retention: Samples With DNA Description:
Samples with DNA: blood, saliva or liver tissue.
|
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| 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. | ||||||||
| Condition |
|
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| Intervention | Not Provided | ||||||||
| Study Groups/Cohorts | Not Provided | ||||||||
| Publications * | Not Provided | ||||||||
|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status | Recruiting | ||||||||
| Estimated Enrollment |
1600 | ||||||||
| Original Estimated Enrollment | Same as current | ||||||||
| Estimated Study Completion Date | June 30, 2029 | ||||||||
| Estimated Primary Completion Date | June 30, 2028 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria |
Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender |
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| Ages | up to 99 Years (Child, Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts |
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| Listed Location Countries | United States | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number | NCT03959800 | ||||||||
| Other Study ID Numbers | STUDY20050018 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement | Not Provided | ||||||||
| Responsible Party | Rakesh Sindhi, University of Pittsburgh | ||||||||
| Study Sponsor | University of Pittsburgh | ||||||||
| Collaborators | Not Provided | ||||||||
| Investigators | Not Provided | ||||||||
| PRS Account | University of Pittsburgh | ||||||||
| Verification Date | June 2021 | ||||||||
