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

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

Status
Not yet recruiting
Phases
Unknown
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT07793994
Enrollment
130
Registered
2026-08-31
Start date
2026-09-01
Completion date
2030-03-31
Last updated
2026-08-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

CNS Tumor, Pediatric Cancer, Refractory Cancer, Relapsed Cancer, Solid Tumor

Keywords

Relapsed/Refractory CNS Tumor, Relapsed/Refractory Solid Tumor

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.

Interventions

DIAGNOSTIC_TESTBulk RNA Sequencing

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

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

Sponsors

B+ Foundation
CollaboratorUNKNOWN
Dana-Farber Cancer Institute
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SCREENING
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
No minimum to 30 Years
Healthy volunteers
No

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.

Design outcomes

Primary

MeasureTime frameDescription
Antibody-Drug Conjugate (ADC) Target Expression Screening Success Rate [Prospective Cohort]Week 6ADC 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.

Secondary

MeasureTime frameDescription
ADC Target Antigen Expression Rate by Bulk RNA Sequencing [Prospective Cohort]Week 4Bulk RNA sequencing is performed on one tumor sample collected from participants with relapsed/refractory CNS or solid tumors. Expression is defined using a predefined threshold of transcripts per million (TPM) ≥3. The ADC target antigen expression rate is the proportion of participants with expression of at least one evaluated ADC target antigen.
ADC Target Antigen Expression Rate by Immunohistochemistry (IHC) [Prospective Cohort]Week 6IHC is performed on one tumor sample collected from participants with relapsed/refractory CNS or solid tumors. Candidate ADC target antigens identified by screening bulk RNA sequencing are evaluated by IHC. Protein expression is assessed using semi-quantitative staining intensity scores (0-3+) and H-scores. The ADC target antigen expression rate is the proportion of participants with expression of at least one evaluated ADC target antigen by IHC.
ADC Target Antigen Transcript Expression Levels by Histologic Diagnosis [Prospective Cohort]Week 4All potential ADC target antigens are evaluated regardless of histologic diagnosis, and transcript expression is quantified using TPM.
ADC Target Antigen Protein Expression Levels by Histologic Diagnosis [Prospective Cohort]Week 6Candidate ADC target antigens identified by screening bulk RNA sequencing are evaluated by IHC. Protein expression is assessed using semi-quantitative staining intensity scores (0-3+) and H-scores, with higher scores indicating greater protein expression
Change in ADC Target Antigen Transcript Expression Level from Diagnosis to Relapse [Prospective Cohort]48 monthsADC target antigen transcript expression is measured by bulk RNA sequencing. Transcript expression is quantified as TPM.
Change in ADC Target Antigen Protein Expression Level from Diagnosis to Relapse [Prospective Cohort]48 monthsADC target antigen protein expression is measured by IHC. Protein expression is assessed using semi-quantitative staining intensity scores (0-3+) and H-scores, with higher scores indicating greater protein expression.
ADC Treatment Rate [Prospective Cohort]48 monthsADC treatment rate is defined as the proportion of participants who receive an ADC as part of their clinical care following return of study results.
ADC Therapy Response Rate [Prospective Cohort]48 monthsADC therapy response rate is defined as the proportion of participants classified as responders according to tumor-specific response criteria. Response is assessed using the International Neuroblastoma Response Criteria (INRC) for neuroblastoma, Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 for extracranial solid tumors, and Response Assessment in Neuro-Oncology (RANO) criteria for primary central nervous system (CNS) tumors.
ADC Therapy Response Rate [Retrospective Cohort]48 monthsADC therapy response rate is defined as the proportion of participants who are classified as responders according to tumor-specific response criteria following ADC therapy.

Countries

United States

Contacts

CONTACTSteven Dubois, MD,MS
Steven_Dubois@dfci.harvard.edu617-632-5460
PRINCIPAL_INVESTIGATORSteven Dubois, MD,MS

Dana-Farber Cancer Institute

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 1, 2026