Thyroid CancerPhase 2NCT05119296
Phase II Trial of Pembrolizumab in Metastatic or Locally Advanced Anaplastic/Undifferentiated Thyroid Cancer
A Phase 2 study testing Pembrolizumab in people with Thyroid Cancer, run by Saad A Khan at 1 site in United States.
Trial summary
This is a single-arm, open-label trial designed to evaluate the activity of pembrolizumab therapy in anaplastic thyroid cancer in patients with no curative alternative therapy. Pembrolizumab (Keytruda-Merck) 200 mg, given IV every 3 weeks, until evidence of progression, intolerance of treatment, withdrawal of consent… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thyroid 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
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Feb 2022to Jul 2027
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Histologically- or cytologically-confirmed diagnosis of anaplastic thyroid cancer (ATC) or undifferentiated thyroid cancer (UTC). A diagnosis of possible ATC/UTC will be allowed if the clinical presentation is consistent with anaplastic or undifferentiated thyroid cancer.
- Disease characteristics one of the following:
- Unresectable ATC/UTC limited to the neck:
- Subjects must have received radiation therapy or surgery to primary tumor and have subsequent evidence of ATC/UTC.
- Metastatic ATC/UTC: either with entirely surgically-removed cancer/metastatic only disease, or with disease in the neck not requiring radiation or surgery to the neck mass
- Measurable disease per RECIST v1.1. Lesions situated in a previously-irradiated area are considered measurable if progression has been demonstrated in such lesions.
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- ≥ 18 years of age.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 (within 7 days prior to the first dose of pembrolizumab).
- Absolute neutrophil count (ANC) ≥ 1500/µL (within 10 days prior to the first dose of pembrolizumab).
- Platelets ≥ 100 000/µL (within 10 days prior to the first dose of pembrolizumab).
- Hemoglobin ≥ 9.0 g/dL or ≥ 5.6 mmol/L (within 10 days prior to the first dose of pembrolizumab). Criteria must be met without erythropoietin dependency and without packed red blood cell (pRBC) transfusion within last 2 weeks.
- Creatinine ≤ 1.5 × ULN OR Measured or calculated creatinine clearance per institutional standard ≥ 30 mL/min for subject with creatinine levels >1.5 × institutional ULN (GFR can also be used in place of creatinine or CrCl) (test within 10 days prior to the first dose of pembrolizumab).
- Total bilirubin ≤ 1.5 × ULN OR Direct bilirubin ≤ ULN for participants with total bilirubin levels >1.5 × ULN (test within 10 days prior to the first dose of pembrolizumab).
- AST (SGOT) and ALT (SGPT) ≤ 2.5 × ULN (≤ 5 × ULN for participants with liver metastases) (test within 10 days prior to the first dose of pembrolizumab).
- International normalized ratio (INR) ≤ 1.5 × ULN OR Prothrombin time (PT) ≤ 1.5 × ULN (Exception: subject is receiving anticoagulant therapy and PT or aPTT is within therapeutic range of intended use of anticoagulants)
- The subject [or legally acceptable representative (LAR) if applicable] has the apparent ability to understand and the willingness to personally sign the written IRB-approved informed consent document.
- Not a woman of childbearing potential (WOCBP), or if WOCBP, not pregnant (negative urine pregnancy test within 72 hours prior to first dose of pembrolizumab), not breastfeeding, and agrees to follow contraceptive guidance per protocol Appendix A. NOTE: if the urine test is positive or cannot be confirmed as negative, a negative serum pregnancy test is required.
Who cannot join
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, CTLA-4, OX-40, CD137).
- Has received prior systemic anti-cancer therapy including investigational agents within 4 weeks prior to study treatment start. Note: Participants must have recovered from all AEs due to previous therapies to ≤ Grade 1 or baseline. Participants with ≤ Grade 2 neuropathy may be eligible. Participants with endocrine-related AEs Grade ≤ 2 requiring treatment or hormone replacement may be eligible. Note: If the participant had major surgery, the participant must have recovered adequately from the procedure and/or any complications from the surgery prior to starting study intervention. Note: Patients who have most recently been treated with dabrafenib and/or trametinib and/or lenvatinib require a washout period of 1 week from their last dose.
- Has received prior radiotherapy within 2 weeks of start of study intervention. Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (≤ 2 weeks of radiotherapy) to non-CNS disease.
- Has received a live vaccine or live-attenuated vaccine within 30 days prior to the first dose of study drug. Administration of killed vaccines is allowed.
- Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 4 weeks prior to the first dose of study intervention. Subject in the follow-up phase of an investigational study may participate as long as it has been ≥4 weeks after the last dose of the previous investigational agent.
- Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 7 days prior to the first dose of study drug.
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- Has a known additional malignancy that is progressing or is requiring active treatment within the past 1 year. Subjects with basal cell carcinoma of the skin, squamous cell carcinoma of the skin or carcinoma in situ (eg, breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
- Has known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, ie, without evidence of progression for at least 4 weeks by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable and without requirement of steroid treatment for at least 14 days prior to first dose of study intervention.
- Has a history of non-infectious pneumonitis/interstitial lung disease that required steroids or has current pneumonitis/interstitial lung disease (ILD).
- Has an active infection requiring systemic therapy.
- Has a known history of Human Immunodeficiency Virus (HIV) infection. Note: HIV testing is not required unless mandated by local health authority.
- Has a known history of Hepatitis B (defined as Hepatitis B surface antigen [HBsAg] reactive) or known active Hepatitis C virus (defined as HCV RNA is detected) infection. Note: no testing for Hepatitis B and Hepatitis C is required unless mandated by local health authority
- Has active autoimmune disease that has required systemic treatment in the past 2 years (ie, with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg, thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc) is not considered a form of systemic treatment and is allowed.
- Has severe hypersensitivity (≥ Grade 3) to pembrolizumab and/or any of its excipients.
- Has other co-morbid disease or intercurrent illness.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the participant's participation for the full duration of the study, or is not in the best interest of the participant 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 study, starting with the screening visit through 120 days after the last dose of trial treatment.
- Has had an allogenic tissue/solid organ transplant.
Trial locations and contacts
1 site in 1 country.
About this study
This is a single-arm, open-label trial designed to evaluate the activity of pembrolizumab therapy in anaplastic thyroid cancer in patients with no curative alternative therapy. Pembrolizumab (Keytruda-Merck) 200 mg, given IV every 3 weeks, until evidence of progression, intolerance of treatment, withdrawal of consent or death
- Conditions
- Thyroid Cancer
- Being tested
- Pembrolizumab
- Medical subject headings
- Thyroid Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabDrug
Also called Keytruda
Pembrolizumab (Keytruda-Merck) 200 mg, given IV every 3 weeks, until evidence of progression, intolerance of treatment, withdrawal of consent or death
| Study group | Role | What they receive |
|---|---|---|
| Pembrolizumab 200 mg | Experimental | Participants will receive pembrolizumab (Keytruda) 200 mg administered by IV infusion every 3 weeks. Participants will remain on study and receive pembrolizumab for the nominal duration of treatment (35 cycles, approximately 2 years) or until there is evidence of disease progression by RECIST, unacceptable toxicity, withdrawal of consent, or discontinuation of the trial for any other reason. Receives: Drug: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (OR)
Overall response (OR) represents those participants that collectively achieve either complete response (CR) or partial response (PR) within 6 months, as defined below per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.
- Complete Response (CR) = Disappearance of all target lesions
- Partial Response (PR) = ≥ 30% decrease in the sum of the longest diameter of target lesions
- Overall Response (OR) = CR + PR
- Progressive disease (PD) = 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s)
- Stable disease (SD) = Small changes that do not meet any of the above criteria The outcome is reported as the cumulative number of participants that achieve either a CR or PR, a number without dispersion.
Measured: 6 months
Secondary outcomes (3)
Progression-free Survival (PFS)
Progression-free survival (PFS) means to remain alive without disease progression. Progressive disease is defined as a 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s). The outcome is reported as the number of participants that remained alive at 2 years after the beginning of treatment (or last available data) without progression, a number without dispersion.
Measured: 2 years
Overall Survival (OS)
Overall survival (OS) means to remain alive without consideration of treatment response status. The outcome is reported as the number of participants that remained alive at 2 years after the beginning of treatment, a number without dispersion. Subjects who become lost-to-follow-up before the 2-year assessment are not included.
Measured: 2 years
Related Adverse Events
To evaluate the association between safety/toxicity outcome, we applied the multivariate logistic regression with Safety of the study treatment pembrolizumab is based on the number of adverse effects caused by pembrolizumab, referred to as related adverse events.
Measured: From first dose through 30 days after last dose, an average of 30 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2021
- Study start15 Feb 2022
- Primary completion16 May 2025
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Thyroid CancerStudies of the same condition, open and under way.
- 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
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Saad A Khan
- Sponsor type
- University, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor-investigator, Saad A Khan, Stanford University
- Investigators
- Saad A Khan, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05119296
- Sponsor's protocol ID
- IRB-61712
- Other IDs
- NCI-2022-05095
- Status last verified
- Aug 2026
- First submitted
- 29 Oct 2021
- Last updated
- 9 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Pembrolizumab in Metastatic or Locally Advanced Anaplastic/Undifferentiated Thyroid Cancer
- ClinicalTrials.gov ID
- NCT05119296
- Acronym / sponsor ID
- IRB-61712
- Conditions
- Thyroid Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Saad A KhanUniversity, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 15 Feb 2022 (actual)
- Primary completion
- 16 May 2025 (actual)
- Study completion
- Jul 2027 (estimated)
- First posted
- 15 Nov 2021 (actual)
- Results
- Posted 9 Sep 2026
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 single-arm, open-label trial designed to evaluate the activity of pembrolizumab therapy in anaplastic thyroid cancer in patients with no curative alternative therapy. Pembrolizumab (Keytruda-Merck) 200 mg, given IV every 3 weeks, until evidence of progression, intolerance of treatment, withdrawal of consent or death
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pembrolizumab 200 mg | Experimental | Drug: PembrolizumabParticipants will receive pembrolizumab (Keytruda) 200 mg administered by IV infusion every 3 weeks. Participants will remain on study and receive pembrolizumab for the nominal duration of treatment (35 cycles, approximately 2 years) or until there is evidence of disease progression by RECIST, unacceptable toxicity, withdrawal of consent, or discontinuation of the trial for any other reason. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Histologically- or cytologically-confirmed diagnosis of anaplastic thyroid cancer (ATC) or undifferentiated thyroid cancer (UTC). A diagnosis of possible ATC/UTC will be allowed if the clinical presentation is consistent with anaplastic or undifferentiated thyroid cancer.
- Disease characteristics one of the following:
- Subjects must have received radiation therapy or surgery to primary tumor and have subsequent evidence of ATC/UTC.
- Metastatic ATC/UTC: either with entirely surgically-removed cancer/metastatic only disease, or with disease in the neck not requiring radiation or surgery to the neck mass
Key exclusion criteria (19 listed)
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, CTLA-4, OX-40, CD137).
- Has received prior systemic anti-cancer therapy including investigational agents within 4 weeks prior to study treatment start. Note: Participants must have recovered from all AEs due to previous therapies to ≤ Grade 1 or baseline. Participants with ≤ Grade 2 neuropathy may be eligible. Participants with endocrine-related AEs Grade ≤ 2 requiring treatment or hormone replacement may be eligible. Note: If the participant had major surgery, the participant must have recovered adequately from the procedure and/or any complications from the surgery prior to starting study intervention. Note: Patients who have most recently been treated with dabrafenib and/or trametinib and/or lenvatinib require a washout period of 1 week from their last dose.
- Has received prior radiotherapy within 2 weeks of start of study intervention. Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (≤ 2 weeks of radiotherapy) to non-CNS disease.
- Has received a live vaccine or live-attenuated vaccine within 30 days prior to the first dose of study drug. Administration of killed vaccines is allowed.
- Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 4 weeks prior to the first dose of study intervention. Subject in the follow-up phase of an investigational study may participate as long as it has been ≥4 weeks after the last dose of the previous investigational agent.
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: KeytrudaPembrolizumab (Keytruda-Merck) 200 mg, given IV every 3 weeks, until evidence of progression, intolerance of treatment, withdrawal of consent or death | Drug | Pembrolizumab 200 mg |
Primary outcome
Overall Response Rate (OR)
Time frame: 6 months
Overall response (OR) represents those participants that collectively achieve either complete response (CR) or partial response (PR) within 6 months, as defined below per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. * Complete Response (CR) = Disappearance of all target lesions * Partial Response (PR) = ≥ 30% decrease in the sum of the longest diameter of target lesions * Overall Response (OR) = CR + PR * Progressive disease (PD) = 20% increase in the sum of the longest diameter of target lesions, and/or the appearance of one or more new lesion(s) * Stable disease (SD) = Small changes that do not meet any of the above criteria The outcome is reported as the cumulative number of participants that achieve either a CR or PR, a number without dispersion.
3 secondary outcomes
Progression-free Survival (PFS)
Time frame: 2 years
Overall Survival (OS)
Time frame: 2 years
Related Adverse Events
Time frame: From first dose through 30 days after last dose, an average of 30 days.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Saad A Khan principal investigator
Stanford University
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 Thyroid Cancer, run by Saad A Khan at 1 site in United States.
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 17 inclusion and 19 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 9 Sep 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 United States. 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?
Yes. Results are posted in the registry (first posted 9 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Response Rate (OR) (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Sep 2026.