CNS TumorNCT07793994
Pedi-STAR: Surface Target Antibody-drug Conjugate (ADC) Antigen Expression in Relapsed/Refractory Pediatric Solid and CNS Tumors
A study testing Bulk RNA Sequencing and Immunohistochemistry (IHC) in people with CNS Tumor and Solid Tumor, run by Dana-Farber Cancer Institute at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- CNS Tumor, Solid Tumor, Relapsed Cancer
- What is being tested?
- Bulk RNA Sequencing and Immunohistochemistry (IHC)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 130 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2026to 31 Mar 2030
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 9Show fewer
- 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.
Who cannot join
- adults who are unable to consent
- pregnant women
- incarcerated individuals
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Steven Dubois, MD,MS contact
United States2 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Steven DuBois, MD, MS, 617-632-5460, Steven_Dubois@dfci.harvard.edu
Steven DuBois, MD, MS
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Steven G. DuBois, MD, MS, 617-632-5460, steven_dubois@dfci.harvard.edu
Steven G. DuBois, MD, MS
About this study
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.
Read the 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.
- Conditions
- CNS Tumor, Solid Tumor, Relapsed Cancer, Refractory Cancer, Pediatric Cancer
- Being tested
- Bulk RNA Sequencing, Immunohistochemistry (IHC)
- Keywords
- Relapsed/Refractory CNS Tumor, Relapsed/Refractory Solid Tumor
- Medical subject headings
- Central Nervous System Neoplasms, Recurrence, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bulk RNA SequencingDiagnostic test
Test performed on tumor tissue to measure antibody-drug conjugate (ADC) target expression
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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Diagnostic Test: Immunohistochemistry (IHC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 6
Secondary outcomes (9)
ADC Target Antigen Expression Rate by Bulk RNA Sequencing [Prospective Cohort]
Bulk 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.
Measured: Week 4
ADC Target Antigen Expression Rate by Immunohistochemistry (IHC) [Prospective Cohort]
IHC 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.
Measured: Week 6
ADC Target Antigen Transcript Expression Levels by Histologic Diagnosis [Prospective Cohort]
All potential ADC target antigens are evaluated regardless of histologic diagnosis, and transcript expression is quantified using TPM.
Measured: Week 4
ADC Target Antigen Protein Expression Levels by Histologic Diagnosis [Prospective Cohort]
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, with higher scores indicating greater protein expression
Measured: Week 6
Change in ADC Target Antigen Transcript Expression Level from Diagnosis to Relapse [Prospective Cohort]
ADC target antigen transcript expression is measured by bulk RNA sequencing. Transcript expression is quantified as TPM.
Measured: 48 months
Change in ADC Target Antigen Protein Expression Level from Diagnosis to Relapse [Prospective Cohort]
ADC 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.
Measured: 48 months
ADC Treatment Rate [Prospective Cohort]
ADC treatment rate is defined as the proportion of participants who receive an ADC as part of their clinical care following return of study results.
Measured: 48 months
ADC Therapy Response Rate [Prospective Cohort]
ADC 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.
Measured: 48 months
ADC Therapy Response Rate [Retrospective Cohort]
ADC therapy response rate is defined as the proportion of participants who are classified as responders according to tumor-specific response criteria following ADC therapy.
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2026
- Study startSep 2026 (estimated)
- Primary completion31 Mar 2029 (estimated)
- Study completion31 Mar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CNS TumorStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 130 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- B+ Foundation
- Responsible party
- Principal investigator, Steven DuBois, MD, Dana-Farber Cancer Institute
- Investigators
- Steven Dubois, MD,MS, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07793994
- Sponsor's protocol ID
- 26-218
- Status last verified
- Aug 2026
- First submitted
- 21 Aug 2026
- Last updated
- 31 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.