An interventional study of Bulk RNA Sequencing and Immunohistochemistry (IHC) in CNS Tumor, Solid Tumor and Relapsed Cancer, sponsored by Dana-Farber Cancer Institute. Not yet recruiting at 2 sites in United States. Open to participants aged Up to 30 Years. Per ClinicalTrials.gov, last updated 2026-08-31.
Sponsored by Dana-Farber Cancer Institute · Not applicable, Interventional, and Screening
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:
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.
699 studies on the registry are indexed under Central Nervous System Neoplasms; 66 are open to participants now.
This study's planned enrollment of 130 is above the median of 35 across 487 interventional studies indexed under Central Nervous System Neoplasms.
Browse Central Nervous System Neoplasms studies →Dana-Farber Cancer Institute is the lead sponsor of 834 studies on the registry; 122 are open to participants now.
Of its 114 completed or terminated interventional studies of FDA-regulated products, 78 (68%) have results posted.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
Inclusion Criteria for Prospective Cohort
Inclusion Criteria for Retrospective Cohort
Exclusion Criteria:
Exclusion Criteria for Prospective Cohort
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Adjuvant and neoadjuvant
Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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)
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)
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
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.
Time frame: Week 6
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.
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 48 months
Plan to share: 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.
Supporting information: Study protocol, Statistical analysis plan
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
No publications or documents are linked to this record.
Start is estimated for Sep 2026; primary completion for Mar 2029, 30 months later.
Of 18 interventional studies with no phase under Central Nervous System Neoplasms, half reached primary completion within 127 months of starting (the median), counting those still running; a quarter within 43 and three quarters within 175. This study's planned 30 months is below that.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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