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

ClinicalTrials.gov ID

NCT07793994

Lead Sponsor

Dana-Farber Cancer InstituteOTHER

Overall Status

Not yet recruiting

Study Type

Interventional

Phase

Not Applicable

Enrollment

130Estimated

Last Update Posted

Aug 31, 2026Actual

Start Date

Sep 2026Estimated

Primary Completion Date

Mar 31, 2029Estimated

Completion Date

Mar 31, 2030Estimated

Official Title

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

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.

Detailed Description

This is a non-therapeutic feasibility study evaluating the prospective measurement of antibody-drug conjugate (ADC) target antigen expression in pediatric patients with relapsed or refractory solid or CNS tumors. The study aims to characterize ADC antigen expression patterns, compare expression at diagnosis and relapse, and describe real-world utilization and outcomes of ADCs in pediatric tumors.

Participants will be enrolled into two cohorts: a prospective cohort and a retrospective cohort.

The prospective cohort will undergo eligibility screening, enrollment, specimen submission, bulk RNA sequencing screening, followed by immunohistochemistry (IHC) of highly expressed targets, and clinical return of IHC results to their enrolling provider.The retrospective cohort will include participants who have received ADC therapy as part of routine clinical care and will undergo specimen submission, targeted IHC testing, and clinical data collection.

It is expected that about 130 people will take part in this research.

B+ Foundation is helping to support this research by providing study funding.

• CNS Tumor
• Solid Tumor
• Relapsed Cancer
• Refractory Cancer
• Pediatric Cancer

• Bulk RNA Sequencing
• Immunohistochemistry (IHC)

Ages Eligible for Study

Child (0-17)Adult (18-64)

Sexes Eligible for Study

All

Accepts Healthy Volunteers

No

Eligibility Criteria

Inclusion Criteria:

Inclusion Criteria for Prospective Cohort

• 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).

Inclusion Criteria for Retrospective Cohort

• 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.

Exclusion Criteria:

Exclusion Criteria for Prospective Cohort

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

Primary Purpose

Screening

Allocation

Non-Randomized

Interventional Model

Parallel Assignment

Masking

None (Open Label)

Lead Sponsor

Dana-Farber Cancer InstituteOTHER

Collaborators

B+ FoundationUNKNOWN

steven_dubois@dfci.harvard.edu617-632-5460

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