Squamous Cell Cancer of Head and NeckPhase 2INSPIRENCT02644369
Study of the Effects of Pembrolizumab in Patients With Advanced Solid Tumors
A Phase 2 study testing Pembrolizumab in people with Squamous Cell Cancer of Head and Neck and Triple Negative Breast Cancer, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
This is a phase 2 study whose main purpose is to evaluate gene changes and immune biomarkers in patients with solid tumors during treatment with pembrolizumab and in relation to response to treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Squamous Cell Cancer of Head and Neck, Triple Negative Breast Cancer, Epithelial Ovarian Cancer
- What is being tested?
- Pembrolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteCanada
- Study dates
- 21 Mar 2016to Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be willing and able to provide written informed consent/assent for the trial.
- Be 18 years of age or older on day of signing informed consent.
- Have histologically or cytologically-documented, locally-advanced, or metastatic solid malignancy that is incurable and has either (a) failed prior standard therapy, (b) for which no standard therapy exists, or (c) standard therapy is not considered appropriate by the patient and treating physician. There is no limit to the number of prior treatment regimens.
- Have one of the following advanced (unresectable and/or metastatic) solid tumor indications:
- Squamous cell cancer of head and neck
- Triple negative breast cancer
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- Epithelial ovarian cancer
- Malignant melanoma
- Advanced solid tumors
- Have measurable disease based on Response Evaluation Criteria In Solid Tumors (RECIST) 1.1.
- Be willing to provide tissue from a newly obtained core or excisional biopsy of a tumor lesion. Patients for whom newly-obtained samples cannot be provided (e.g. inaccessible or patient safety concern) will not be eligible for this study.
- Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) Performance Scale.
- Demonstrate adequate organ function.
- Negative pregnancy test for female patients of childbearing potential.
- Must use approved methods of birth control during the course of the study and for 120 days after the last dose of study drug.
Who cannot join
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known history of active TB (Bacillus Tuberculosis).
- Hypersensitivity to pembrolizumab or any of its excipients.
- Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study Day 1 or who has not recovered from adverse events due to agents administered more than 4 weeks earlier.
- Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to Study Day 1 or who has not recovered from adverse events due to a previously administered agent.
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- Has a known additional malignancy that is progressing or requires active treatment.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis.
- Has active autoimmune disease that has required systemic treatment in the past 2 years
- Has known history of, or any evidence of active, non-infectious pneumonitis.
- Has an active infection requiring systemic therapy.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the patient's participation for the full duration of the trial, or is not in the best interest of the patient to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with screening visit through 120 days after the last dose of trial treatment.
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.
- Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies).
- Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
- Has received a live vaccine within 30 days of planned start of study therapy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Investigator-initiated Phase II Study of Pembrolizumab Immunological Response Evaluation
This is a phase 2 study whose main purpose is to evaluate gene changes and immune biomarkers in patients with solid tumors during treatment with pembrolizumab and in relation to response to treatment.
Pembrolizumab is a monoclonal antibody that is designed to block a protein called programmed cell death 1 ligand 1 (PD-L1) which will allow the body's immune system to kill the cancer cells.
Read the detailed description
This study will involve treatment with pembrolizumab, tests and procedures done for safety, and the collection of archival tumor tissue, fresh tumor biopsies, and blood samples for biomarker research (including genetic testing).
- Conditions
- Squamous Cell Cancer of Head and Neck, Triple Negative Breast Cancer, Epithelial Ovarian Cancer, Malignant Melanoma, Advanced Solid Tumors
- Being tested
- Pembrolizumab
- Medical subject headings
- Triple Negative Breast Neoplasms, Carcinoma, Ovarian Epithelial, Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabBiological
Also called Keytruda(TM), MK-3475
Pembrolizumab is a potent and highly selective humanized monoclonal antibody (mAb) of the IgG4/kappa isotype designed to directly block the interaction between PD-1 and its ligands, PD-L1 and PD-L2.
| Study group | Role | What they receive |
|---|---|---|
| Pembrolizumab | Experimental | Pembrolizumab will be given by intravenous infusion (IV, given by vein) at a dose of 200 mg, once every 3 weeks. Receives: Biological: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in genomic and immune biomarkers that will be measured in blood and tumor pre-treatment, on-treatment and at progression (progression biopsies only done for those who have complete or partial responses, or stable disease for more than 4 months)
T-test or Wilcoxon's test
Measured: 5 years
Secondary outcomes (9)
Overall response rate
summary statistics
Measured: 5 years
Changes in circulating tumor DNA genomic biomarkers
T-test or Wilcoxon's test
Measured: 5 years
Changes in radiomic imaging parameters
T-test or Wilcoxon's test
Measured: 5 years
Correlation between tumor genomic profiles and radiomic imaging signatures
Pearson correlation coefficients, chi-square tests, one- or two-sample t-tests or logistic regression analyses as appropriate. Non-parametric tests such as Spearman correlation coefficients, Fisher's exact tests and Wilcoxon rank sum tests may be substituted if necessary.
Measured: 5 years
Changes in immune cell subsets in the blood and tumor microenvironment
T-test or Wilcoxon's test
Measured: 5 years
Positive predictive value and negative predictive value of in vitro predictive assay for Pembrolizumab response
Positive predictive value and negative predictive value
Measured: 5 years
Distribution of drug tumor penetration using a mass spectrometry assay
summary statistics
Measured: 5 years
Baseline tumor RNA expression profile for immune inhibitory genes
summary statistics
Measured: 5 years
Comparison of baseline tumor RNA expression profile for immune inhibitory genes with clinical outcome (response)
T-test or Wilcoxon's test
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2015 (estimated)
- Study start21 Mar 2016
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Squamous Cell Cancer of Head and NeckStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor
- Investigators
- Lillian Siu, M.D., Principal investigator, Princess Margaret Cancer Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02644369
- Sponsor's protocol ID
- INSPIRE-001
- Acronym
- INSPIRE
- Status last verified
- Sep 2026
- First submitted
- 27 Oct 2015
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Mayerhoefer ME, Kienzle A, Woo S, Vargas HA. Update on Liquid Biopsy. Radiology. 2025 Jun;315(3):e241030. doi: 10.1148/radiol.241030.
PubMed 40525978Hernando-Calvo A, Yang SYC, Vila-Casadesus M, Han M, Liu ZA, Berman AHK, Spreafico A, Razak AA, Lheureux S, Hansen AR, Lo Giacco D, Abbas-Aghababazadeh F, Matito J, Haibe-Kains B, Pugh TJ, Bratman SV, Aleshin A, Berche R, Saavedra O, Garralda E, Elston S, Siu LL, Ohashi PS, Vivancos A, Bedard PL. Combined Transcriptome and Circulating Tumor DNA Longitudinal Biomarker Analysis Associates With Clinical Outcomes in Advanced Solid Tumors Treated With Pembrolizumab. JCO Precis Oncol. 2024 Aug;8:e2400100. doi: 10.1200/PO.24.00100.
PubMed 39178369Koppensteiner L, Mathieson L, Pattle S, Dorward DA, O'Connor R, Akram AR. Location of CD39+ T cell subpopulations within tumors predict differential outcomes in non-small cell lung cancer. J Immunother Cancer. 2023 Aug;11(8):e006770. doi: 10.1136/jitc-2023-006770.
PubMed 37648263Bratman SV, Yang SYC, Iafolla MAJ, Liu Z, Hansen AR, Bedard PL, Lheureux S, Spreafico A, Razak AA, Shchegrova S, Louie M, Billings P, Zimmermann B, Sethi H, Aleshin A, Torti D, Marsh K, Eagles J, Cirlan I, Hanna Y, Clouthier DL, Lien SC, Ohashi PS, Xu W, Siu LL, Pugh TJ. Personalized circulating tumor DNA analysis as a predictive biomarker in solid tumor patients treated with pembrolizumab. Nat Cancer. 2020 Sep;1(9):873-881. doi: 10.1038/s43018-020-0096-5. Epub 2020 Aug 3.
PubMed 35121950Iafolla MAJ, Yang C, Chandran V, Pintilie M, Li Q, Bedard PL, Hansen A, Lheureux S, Spreafico A, Razak AA, Hakgor S, Giesler A, Pugh TJ, Siu LL. Predicting Toxicity and Response to Pembrolizumab Through Germline Genomic HLA Class 1 Analysis. JNCI Cancer Spectr. 2020 Dec 29;5(1):pkaa115. doi: 10.1093/jncics/pkaa115. eCollection 2021 Feb.
PubMed 33554038
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigator-initiated Phase II Study of Pembrolizumab Immunological Response Evaluation
- ClinicalTrials.gov ID
- NCT02644369
- Acronym / sponsor ID
- INSPIRE / INSPIRE-001
- Conditions
- Squamous Cell Cancer of Head and Neck, Triple Negative Breast Cancer, Epithelial Ovarian Cancer, Malignant Melanoma, Advanced Solid Tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 21 Mar 2016 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 31 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2 study whose main purpose is to evaluate gene changes and immune biomarkers in patients with solid tumors during treatment with pembrolizumab and in relation to response to treatment.
Pembrolizumab is a monoclonal antibody that is designed to block a protein called programmed cell death 1 ligand 1 (PD-L1) which will allow the body's immune system to kill the cancer cells.
Detailed description
This study will involve treatment with pembrolizumab, tests and procedures done for safety, and the collection of archival tumor tissue, fresh tumor biopsies, and blood samples for biomarker research (including genetic testing).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pembrolizumab | Experimental | Biological: PembrolizumabPembrolizumab will be given by intravenous infusion (IV, given by vein) at a dose of 200 mg, once every 3 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Be willing and able to provide written informed consent/assent for the trial.
- Be 18 years of age or older on day of signing informed consent.
- Have histologically or cytologically-documented, locally-advanced, or metastatic solid malignancy that is incurable and has either (a) failed prior standard therapy, (b) for which no standard therapy exists, or (c) standard therapy is not considered appropriate by the patient and treating physician. There is no limit to the number of prior treatment regimens.
- Have one of the following advanced (unresectable and/or metastatic) solid tumor indications:
- Have measurable disease based on Response Evaluation Criteria In Solid Tumors (RECIST) 1.1.
Key exclusion criteria (18 listed)
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known history of active TB (Bacillus Tuberculosis).
- Hypersensitivity to pembrolizumab or any of its excipients.
- Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study Day 1 or who has not recovered from adverse events due to agents administered more than 4 weeks earlier.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PembrolizumabAlso: Keytruda(TM), MK-3475Pembrolizumab is a potent and highly selective humanized monoclonal antibody (mAb) of the IgG4/kappa isotype designed to directly block the interaction between PD-1 and its ligands, PD-L1 and PD-L2. | Biological | Pembrolizumab |
Primary outcome
Changes in genomic and immune biomarkers that will be measured in blood and tumor pre-treatment, on-treatment and at progression (progression biopsies only done for those who have complete or partial responses, or stable disease for more than 4 months)
Time frame: 5 years
T-test or Wilcoxon's test
9 secondary outcomes
Overall response rate
Time frame: 5 years
Changes in circulating tumor DNA genomic biomarkers
Time frame: 5 years
Changes in radiomic imaging parameters
Time frame: 5 years
Correlation between tumor genomic profiles and radiomic imaging signatures
Time frame: 5 years
Changes in immune cell subsets in the blood and tumor microenvironment
Time frame: 5 years
Positive predictive value and negative predictive value of in vitro predictive assay for Pembrolizumab response
Time frame: 5 years
Distribution of drug tumor penetration using a mass spectrometry assay
Time frame: 5 years
Baseline tumor RNA expression profile for immune inhibitory genes
Time frame: 5 years
Comparison of baseline tumor RNA expression profile for immune inhibitory genes with clinical outcome (response)
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Derived from this study: PubMed 40525978Mayerhoefer ME, Kienzle A, Woo S, Vargas HA. Update on Liquid Biopsy. Radiology. 2025 Jun;315(3):e241030. doi: 10.1148/radiol.241030.
- Derived from this study: PubMed 39178369Hernando-Calvo A, Yang SYC, Vila-Casadesus M, Han M, Liu ZA, Berman AHK, Spreafico A, Razak AA, Lheureux S, Hansen AR, Lo Giacco D, Abbas-Aghababazadeh F, Matito J, Haibe-Kains B, Pugh TJ, Bratman SV, Aleshin A, Berche R, Saavedra O, Garralda E, Elston S, Siu LL, Ohashi PS, Vivancos A, Bedard PL. Combined Transcriptome and Circulating Tumor DNA Longitudinal Biomarker Analysis Associates With Clinical Outcomes in Advanced Solid Tumors Treated With Pembrolizumab. JCO Precis Oncol. 2024 Aug;8:e2400100. doi: 10.1200/PO.24.00100.
- Derived from this study: PubMed 37648263Koppensteiner L, Mathieson L, Pattle S, Dorward DA, O'Connor R, Akram AR. Location of CD39+ T cell subpopulations within tumors predict differential outcomes in non-small cell lung cancer. J Immunother Cancer. 2023 Aug;11(8):e006770. doi: 10.1136/jitc-2023-006770.
- Derived from this study: PubMed 35121950Bratman SV, Yang SYC, Iafolla MAJ, Liu Z, Hansen AR, Bedard PL, Lheureux S, Spreafico A, Razak AA, Shchegrova S, Louie M, Billings P, Zimmermann B, Sethi H, Aleshin A, Torti D, Marsh K, Eagles J, Cirlan I, Hanna Y, Clouthier DL, Lien SC, Ohashi PS, Xu W, Siu LL, Pugh TJ. Personalized circulating tumor DNA analysis as a predictive biomarker in solid tumor patients treated with pembrolizumab. Nat Cancer. 2020 Sep;1(9):873-881. doi: 10.1038/s43018-020-0096-5. Epub 2020 Aug 3.
- Derived from this study: PubMed 33554038Iafolla MAJ, Yang C, Chandran V, Pintilie M, Li Q, Bedard PL, Hansen A, Lheureux S, Spreafico A, Razak AA, Hakgor S, Giesler A, Pugh TJ, Siu LL. Predicting Toxicity and Response to Pembrolizumab Through Germline Genomic HLA Class 1 Analysis. JNCI Cancer Spectr. 2020 Dec 29;5(1):pkaa115. doi: 10.1093/jncics/pkaa115. eCollection 2021 Feb.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lillian Siu, M.D. principal investigator
Princess Margaret Cancer Centre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pembrolizumab in people with Squamous Cell Cancer of Head and Neck and Triple Negative Breast Cancer, run by University Health Network, Toronto at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in genomic and immune biomarkers that will be measured in blood and tumor pre-treatment, on-treatment and at progression (progression biopsies only done for those who have complete or partial responses, or stable disease for more than 4 months) (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02644369, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.