Feasibility of Screening for Early Late Effects of Contemporary Therapy in High-Risk Neuroblastoma Survivors

LEGACY (Late Effects of GD2 Antibody and Chemotherapy in Youths): Feasibility of Screening for Early Late Effects of Contemporary Therapy in High-Risk Neuroblastoma Survivors

The study participant is being asked to be in a research study called LEGACY, because you were treated for high-risk neuroblastoma (HR-NBL).

Primary Objective

To determine the feasibility of conducting comprehensive evaluations, leveraging the established SJLIFE (St. Jude Lifetime Cohort Study) study infrastructure, in survivors of HR-NBL (high-risk neuroblastoma) who are greater than 2 years from completion of contemporary therapy and were previously treated at SJCRH (St. Jude Children's Research Hospital).

Exploratory Objectives

• To describe the health outcomes of survivors of HR-NBL previously treated at SJCRH with contemporary era therapy who are greater than 2 years from completion of therapy.
• To describe the relationship between patient outcomes and social determinants of health (SDOH) in survivors of HR-NBL previously treated at SJCRH with contemporary era therapy who are greater than 2 years from completion of therapy.

Study Overview

• Status: Active, not recruiting
• Conditions: High-risk Neuroblastoma

Detailed Description

The study participant will have blood tests, tests to measure how your nervous system and stomach works, and an ultrasound of your liver for this study. The researchers will test how well your heart and blood vessels are working.

• Study Type: Observational
• Enrollment (Actual): 34

Contacts and Locations

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All participants who meet eligibility criteria and consent to enrollment on the study.

Description

Inclusion Criteria:

• Participants were diagnosed with neuroblastoma at < 18 years of age and categorized as high-risk at that time, according to INSS26 or INRGSS27 criteria.
• Participants were treated for HR-NBL at SJCRH on NB2012 or as per NB2012.
• Participants are ≥ 2 years from completion of therapy. Participants are 5 years of age or older.
• Participant or legal guardian is able and willing to give informed consent.
• Participants may choose to participate in all or a fraction of the proposed assessments; refusal to participate in some aspects of the study will not preclude their inclusion.
• Participants must also complete enrollment on SJLIFE.

Exclusion Criteria:

• Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
• Participants who had relapsed or refractory disease during or following completion of modern era treatment for HR-NBL.
• Participants who are pregnant or breastfeeding.
• Participants who are unable to read and understand English

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility will be measured by at least 66% participation rate	To determine the feasibility of conducting comprehensive evaluations, leveraging the established SJLIFE (St. Jude Lifetime Cohort Study) study infrastructure, in survivors of HR-NBL (high-risk neuroblastoma) who are greater than 2 years from completion of contemporary therapy and were previously treated at SJCRH (St. Jude Children's Research Hospital). To establish the feasibility, we will estimate the participation rate. The study is considered to have an acceptable participation rate if the 95% lower bound of the participation rate exceeds 66%. Participation is defined as completing ≥ 75% of all proposed assessments.	Up to 24 months after participant enrollment.
To describe the health outcomes of survivors of HR-NBL previously treated at SJCRH with contemporary era therapy who are greater than 2 years from completion of therapy.	Participants will go through a comprehensive assessment including the SJLIFE core battery and additional studies in the categories of neurological and autonomic, immunological, quality of life, hematological, renal and metabolic, endocrine, cardiorespiratory, and gastrointestinal (see table below). Their health conditions will be defined by normative data and will be graded using the Revised Common Terminology Criteria for Adverse Events (CTCAE) used by Hudson et al. in SJLIFE studies. The prevalence, cumulative incidence, and latency of selected treatment complications in survivors will be estimated. Resting/minimum/maximum heart rate, nerve velocities (median, deep peroneal, common peroneal, Sural), sweat total/baseline/end offset volumes, complete blood cou	Health outcomes will be assessed on Days 1, 2, 3, 4, or 5 during the SJLIFE and LEGACY campus visit.
Socioeconomic status will be measured by self-reported demographic information	Answers to the household survey, which includes single-item evaluations of self-reported race, ethnicity and primary language, collected per U.S. Health and Human Services guidelines, health literacy utilizing a validated 1-item screen, educational attainment, household materials hardship (HMH), and household income.	Socioeconomic status, assessed via the household survey, will be assessed on Days 1, 2, 3, 4, or 5 during the LEGACY and SJLIFE campus visit.

Collaborators and Investigators

• Sponsor: St. Jude Children's Research Hospital
• Investigators: Principal Investigator: Sara Federico, MD, St. Jude Children's Research Hospital

Publications and helpful links

Helpful Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Study record dates

Study Major Dates

• Study Start (Actual): October 21, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: June 14, 2024
• First Submitted That Met QC Criteria: June 24, 2024
• First Posted (Actual): June 28, 2024

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neuroblastoma
• Other Study ID Numbers: LEGACY; NCI-2024-05183 (Registry Identifier: NCI Clinical Trial Registration Program)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No