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

• ClinicalTrials.gov Identifier: NCT03686566
• Recruitment Status: Recruiting
• First Posted: September 27, 2018
• Last Update Posted: August 6, 2021
• Sponsor: Tanya Watt
• Information provided by (Responsible Party): Tanya Watt, University of Texas Southwestern Medical Center

Tracking Information

• First Submitted Date: September 10, 2018
• First Posted Date: September 27, 2018
• Last Update Posted Date: August 6, 2021
• Actual Study Start Date: November 16, 2018
• Estimated Primary Completion Date: November 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 30, 2021)

• Metabolic phenotypes [ Time Frame: 2 years ]
  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)
• Compare the metabolic phenotype with the result of histopathological diagnosis and genetic alterations of the specific tumor [ Time Frame: 2 years ]
  We will collect data and information from the patient's medical record including pathologic diagnosis and genetic testing results throughout their treatment

Original Primary Outcome Measures (submitted: September 24, 2018)

• metabolites [ Time Frame: 2 years ]
  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)
• Compare the metabolic phenotype with the result of histopathological diagnosis and genetic alterations of the specific tumor [ Time Frame: 2 years ]
  We will collect data and information from the patient's medical record including pathologic diagnosis and genetic testing results throughout their treatment

Current Secondary Outcome Measures (submitted: July 30, 2021)

• Metabolic evolution of tumors over time [ Time Frame: 2 years ]
  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)
• Metabolic change due to chemotherapy [ Time Frame: 2 years ]
  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.
• Metabolic phenotypes and outcomes [ Time Frame: 2 years ]
  Assess for correlations between metabolism and patient outcome if applicable

Original Secondary Outcome Measures (submitted: September 24, 2018)

• metabolic evolution [ Time Frame: 2 years ]
  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)
• metabolic change due to chemotherapy [ Time Frame: 2 years ]
  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.
• outcomes [ Time Frame: 2 years ]
  Assess for correlations between metabolism and patient outcome if applicable
• accuracy of sample results (whether the metabolic signatures can be detected) using a 25% dose reduction. (compared to STU062010-157) [ Time Frame: 2 years ]
  As the prior study used less sensitive measuring devices as compared to the tools available to study metabolism now, we will attempt a dose reduction and see if the results are comparable. if not, we will use the prior dosing regimen.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Pediatric Solid Tumor Metabolism [A Prospective Study Exploring Metabolism of Solid Tumors in Pediatrics]
• Official Title: Pediatric Solid Tumor Metabolism [A Prospective, Single Center Study Exploring Solid Tumor Metabolism of Extra-cranial Tumors in the Pediatric Population]
• Brief Summary: 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.

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
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.

Condition

• Neuroblastoma
• Sarcoma
• Pediatric Cancer

Intervention

• 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 Name: U-C glucose
• Other: No glucose infusion
  Only includes standard pre-operation nursing care

Study Groups/Cohorts

• 13C-glucose infusion
  Tumor samples of patients who receive the optional 13C-glucose infusion will be studied using flux analysis and metabolomic profiling.
  Intervention: Drug: 13C-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.
  Intervention: Other: No glucose infusion

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: July 2, 2021): 100
• Original Estimated Enrollment (submitted: September 24, 2018): 40
• Estimated Study Completion Date: November 2022
• Estimated Primary Completion Date: November 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 26 Years (Child, Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Tanya Watt, MD; 214-456-6363; tanya.watt@utsouthwestern.edu

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03686566
• Other Study ID Numbers: STU 052018-100
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• 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

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Tanya Watt, University of Texas Southwestern Medical Center
• Study Sponsor: Tanya Watt
• Collaborators: Not Provided

Investigators

• Principal Investigator: Tanya Watt, MD; UTSW

• PRS Account: University of Texas Southwestern Medical Center
• Verification Date: July 2021