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To explore metabolic phenotypes of children with extra-cranial solid tumors and compare these with their histopathological and genetic alterations to discover potential novel biomarkers and therapeutic targets to improve outcomes in children with high risk disease.
The principal objective of this study is the metabolic characterization of pediatric solid tumors, with a particular focus on neuroblastoma (NBL) and fusion positive sarcoma (FPS), which will allow the detection of tumor specific metabolic alterations that can be exploited with the aim of developing novel therapeutic strategies and biomarkers.
Cellular metabolism studies provide insight, in a complementary way to genomics, into processes acting downstream from oncogenes and oncogenic fusion proteins, and such insight may point toward previously unrecognized therapeutic targets or onco-metabolites that are traceable as robust biomarkers for response. The investigator's new approach to use an in-vivo comprehensive analysis of metabolic reprograming in FPS/NBL has never been performed in childhood FPS/NBL and will complement genomics studies for these cancers. For this study, the investigators plan to obtain tumor samples at time of surgical biopsy/resection and study their metabolic signatures.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 13C-glucose infusion | Tumor samples of patients who receive the optional 13C-glucose infusion will be studied using flux analysis and metabolomic profiling. |
| |
| No 13C-glucose infusion | Tumor samples of patients who do not choose to receive the optional 13C-glucose infusion will be studied using metabolomic profiling alone. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 13C-glucose | Drug | Includes standard pre-operation nursing care, 13C-glucose infusion and finger stick/IV glucose checks and storage of blood sample approximately every 30 minutes |
| Measure | Description | Time Frame |
|---|---|---|
| Metabolic phenotypes | Upon collection of tumor samples, they will be processed and analyzed with mass spectrometry to learn how the tumor processes the labeled glucose by assessing enrichment of metabolites to identify the active metabolic pathways in each tumor (metabolic phenotype) | 2 years |
| Compare the metabolic phenotype with the result of histopathological diagnosis and genetic alterations of the specific tumor | We will collect data and information from the patient's medical record including pathologic diagnosis and genetic testing results throughout their treatment | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Metabolic evolution of tumors over time | Compare tumor metabolism at different points in therapy (diagnosis, metastasis, recurrence) if the family consents to further studies as their child's condition progresses. Will compare high risk samples to low risk samples within a diagnosis (IE: high risk neuroblastoma vs low risk neuroblastoma) | 2 years |
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Inclusion Criteria:
Exclusion Criteria:
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Children, adolescents and young adults through the age of 26 years old with a suspected malignant extra-cranial solid tumor.
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| Name | Affiliation | Role |
|---|---|---|
| Tanya Watt, MD | UTSW | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UT Southwestern | Dallas | Texas | 75235 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33860280 | Derived | Johnston K, Pachnis P, Tasdogan A, Faubert B, Zacharias LG, Vu HS, Rodgers-Augustyniak L, Johnson A, Huang F, Ricciardo S, Zhao Z, Mathews TP, Watt T, Leavey P, DeBerardinis RJ. Isotope tracing reveals glycolysis and oxidative metabolism in childhood tumors of multiple histologies. Med. 2021 Apr 9;2(4):395-410. doi: 10.1016/j.medj.2021.01.002. Epub 2021 Feb 23. |
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Jul 29, 2025 | |
| Reset | Aug 13, 2025 | |
| Release | Sep 16, 2025 | |
| Reset | Oct 3, 2025 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jul 29, 2025 | Aug 13, 2025 | |||
| Sep 16, 2025 |
| ID | Term |
|---|---|
| D009447 | Neuroblastoma |
| D012509 | Sarcoma |
| D009369 | Neoplasms |
| ID | Term |
|---|---|
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018242 | Neuroectodermal Tumors, Primitive |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
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| No glucose infusion | Other | Only includes standard pre-operation nursing care |
|
| Metabolic change due to chemotherapy | Compare tumor metabolism at different points in therapy (before vs after chemotherapy is given) if the family consents to further studies as their child's condition progresses. For example, tumor sample at time of neuroblastoma biopsy to resection a few months later prior to bone marrow transplantation. | 2 years |
| Metabolic phenotypes and outcomes | Assess for correlations between metabolism and patient outcome if applicable | 2 years |
| Oct 3, 2025 |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |