Pediatric Solid Tumor Metabolism [A Prospective Study Exploring Metabolism of Solid Tumors in Pediatrics]

Pediatric Solid Tumor Metabolism [A Prospective, Single Center Study Exploring Solid Tumor Metabolism of Extra-cranial Tumors in the Pediatric Population]

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.

Study Overview

• Status: Completed
• Conditions: Sarcoma; Neuroblastoma; Pediatric Cancer
• Intervention / Treatment: Drug: 13C-glucose; Other: No glucose infusion

Detailed Description

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.

• Study Type: Observational
• Enrollment (Actual): 84

Contacts and Locations

• Study Contact: Name: Tanya Watt, MD; Phone Number: 214-456-6363; Email: tanya.watt@utsouthwestern.edu

Study Locations

• United States
  • Texas
    • Dallas, Texas, United States, 75235
      • UT Southwestern

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 26 years (Child, Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Children, adolescents and young adults through the age of 26 years old with a suspected malignant extra-cranial solid tumor.

Description

Inclusion Criteria:

• Suspected malignancy
• Age ≤ 26 years and being cared for at Children's Medical Center
• Ability to undergo standard of care diagnostic procedure, including biopsy or resection of the tumor, in the OR or IR at CMC

Exclusion Criteria:

• Poorly controlled diabetes
• Any other medical condition that prevents administration of glucose

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
13C-glucose infusion; Tumor samples of patients who receive the optional 13C-glucose infusion will be studied using flux analysis and metabolomic profiling.	Drug: 13C-glucose; Includes standard pre-operation nursing care, 13C-glucose infusion and finger stick/IV glucose checks and storage of blood sample approximately every 30 minutes; Other Names: U-C glucose
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.	Other: No glucose infusion; Only includes standard pre-operation nursing care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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
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

Collaborators and Investigators

• Sponsor: University of Texas Southwestern Medical Center
• Investigators: Principal Investigator: Tanya Watt, MD, UTSW

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): November 16, 2018
• Primary Completion (Actual): January 2, 2024
• Study Completion (Actual): January 26, 2024

Study Registration Dates

• First Submitted: September 10, 2018
• First Submitted That Met QC Criteria: September 24, 2018
• First Posted (Actual): September 27, 2018

Study Record Updates

• Last Update Posted (Actual): February 28, 2024
• Last Update Submitted That Met QC Criteria: February 26, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma
• Other Study ID Numbers: STU 052018-100

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No