Pedi-STAR: Surface Target Antibody-drug Conjugate (ADC) Antigen Expression in Relapsed/Refractory Pediatric Solid and CNS Tumors

NCT07793994 | Not Yet Recruiting FDA Device

• 1 other identifier: 26-218
• Study Type: interventional
• Target: 130
• Locations: 1 country
• Sites: 2

Brief Summary

This study is looking to learn whether a tumor testing approach can feasibly measure antibody-drug conjugate (ADC) target proteins in children and young adults with relapsed or refractory solid tumors or central nervous system (CNS) tumors. The tumor testing approach is a two-step tumor profiling process of:

• Screening with bulk RNA sequencing to see which ADC target genes are turned on in the tumor.
• Confirmatory immunohistochemistry (IHC) for targets that show positive expression on the RNA screen, to confirm protein expression.

Conditions

CNS Tumor; Solid Tumor; Relapsed Cancer; Refractory Cancer; Pediatric Cancer

Keywords

Relapsed/Refractory CNS Tumor; Relapsed/Refractory Solid Tumor

Outcome Measures

Primary Outcomes (1)

• Antibody-Drug Conjugate (ADC) Target Expression Screening Success Rate [Prospective Cohort]

  ADC target expression screening success rate is defined as the proportion of participants who successfully complete bulk RNA sequencing with interpretable results. If immunohistochemistry (IHC) testing is indicated based on the bulk RNA sequencing results, the participant must also successfully complete IHC testing with interpretable results. If IHC testing is not indicated, no further testing is required. Participants who submit a tissue specimen but do not meet these criteria will be classified as screening failures.

  Week 6

Secondary Outcomes (9)

• ADC Target Antigen Expression Rate by Bulk RNA Sequencing [Prospective Cohort]

  Week 4

• ADC Target Antigen Expression Rate by Immunohistochemistry (IHC) [Prospective Cohort]

  Week 6

• ADC Target Antigen Transcript Expression Levels by Histologic Diagnosis [Prospective Cohort]

  Week 4

• ADC Target Antigen Protein Expression Levels by Histologic Diagnosis [Prospective Cohort]

  Week 6

• Change in ADC Target Antigen Transcript Expression Level from Diagnosis to Relapse [Prospective Cohort]

  48 months

Study Arms (2)

Prospective Cohort

OTHER

Participants with relapsed/refractory solid or CNS tumors will undergo eligibility screening, specimen submission, bulk RNA sequencing, and confirmatory IHC testing. IHC results will be returned to the enrolling provider

Diagnostic Test: Bulk RNA Sequencing; Diagnostic Test: Immunohistochemistry (IHC)

Retrospective Cohort

OTHER

Participants who have received ADC therapy as part of routine clinical care will submit specimens for targeted IHC testing and clinical data collection. No RNA sequencing will be performed, and IHC results will not be returned

Diagnostic Test: Immunohistochemistry (IHC)

Interventions

Bulk RNA Sequencing DIAGNOSTIC_TEST

Test performed on tumor tissue to measure antibody-drug conjugate (ADC) target expression

Prospective Cohort

Immunohistochemistry (IHC) DIAGNOSTIC_TEST

Test performed on tumor tissue to confirm protein expression of antibody-drug conjugate (ADC) target antigens identified by bulk RNA sequencing

Prospective Cohort; Retrospective Cohort

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 0 - 30 years at time of initial diagnosis of the solid or CNS tumor
• Diagnosis of relapsed or refractory solid or CNS tumor, without a limit on the number of prior episodes of relapse. Refractory disease is defined as disease that progresses on standard-of-care upfront therapy or requires salvage therapy due to inadequate response, e.g. bridging chemoimmunotherapy in a patient with high-risk neuroblastoma and poor end-induction response (PEIR).
• Life expectancy of at least 12 weeks at the time of enrollment
• Available specimens Tissue block or unstained slides available expected to yield a minimum of 30 total slides., OR other specimens expected to result in adequate (1) RNA quantity and quality for sequencing and (2) IHC testing may substitute for tissue requirement above with the approval of the PI or study staff designee approval prior to submission enrollment. Tumor tissue from relapse/progression is preferred, but tumor tissue from diagnosis if obtained prior to neoadjuvant chemotherapy is acceptable if no relapse/progression sample available.Note that this requirement may be reduced to 15 total slides for patients who have already had RNA sequencing performed on their tumor material.
• Informed consent for participation for adult participants or parental permission for minor participants (with assent as appropriate based on age of participant).
• Age 0 - 30 years at time of initial diagnosis of the solid or CNS tumor
• Diagnosis of relapsed or refractory solid or CNS tumor, without a limit on the number of prior episodes of relapse. Refractory disease is defined as disease that progresses on standard-of-care upfront therapy or requires salvage therapy due to inadequate response, e.g. bridging chemoimmunotherapy in a patient with high-risk neuroblastoma and poor end-induction response (PEIR).
• Available specimens Tissue block or unstained slides available expected to yield a minimum of 15 total slides OR other specimens expected to result in adequate IHC testing may substitute for tissue above with the approval of the PI or study staff approval designee prior to submission.. Tumor tissue from relapse/progression is preferred, but tumor tissue from diagnosis if obtained prior to neoadjuvant chemotherapy is acceptable if no relapse/progression sample available.
• Prior receipt of an antibody-drug conjugate (ADC). The ADC may have been received via either commercial supply or as a participant on a clinical trial. The ADC may have been received at any time during a patient's treatment course for relapsed or refractory disease and on or after January 1st, 2011 up to the time of enrollment of the final prospective participant.

You may not qualify if:

• adults who are unable to consent
• pregnant women
• incarcerated individuals

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead
• B+ Foundation: collaborator

Study Sites (2)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Central Nervous System Neoplasms; Recurrence; Neoplasms

Interventions

Immunohistochemistry

Condition Hierarchy (Ancestors)

Nervous System Neoplasms; Neoplasms by Site; Nervous System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Histocytochemistry; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Histological Techniques; Investigative Techniques; Immunologic Techniques

Study Officials

• Steven Dubois, MD,MS

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Steven Dubois, MD,MS

CONTACT

617-632-5460; Steven_Dubois@dfci.harvard.edu

Steven Dubois, MD,MS

CONTACT

617-632-5460

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SCREENING
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 21, 2026
• First Posted: August 31, 2026
• Study Start: September 1, 2026
• Primary Completion (Estimated): March 31, 2029
• Study Completion (Estimated): March 31, 2030
• Last Updated: August 31, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu

Locations

US (2)