Stage III Thymoma AJCC v8Phase 1NCT03295227
Pembrolizumab in Treating Participants With Unresectable Thymoma or Thymic Cancer
A Phase 1 study testing Pembrolizumab in people with Stage III Thymoma AJCC v8 and Stage IIIA Thymoma AJCC v8, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase I trial studies the sides effects and best dose of pembrolizumab in treating participants with thymoma or thymic cancer that cannot be removed by surgery. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stage III Thymoma AJCC v8, Stage IIIA Thymoma AJCC v8, Stage IIIB Thymoma AJCC v8
- 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
- 37 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Dec 2017to 31 Dec 2027
- Phase
- Phase 1
- 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
- Unresectable thymoma or thymic carcinoma.
- Any line of prior therapy allowed.
- Be willing and able to provide written informed consent/assent for the trial.
- 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. Newly-obtained is defined as a specimen obtained up to 6 weeks (42 days) prior to initiation of treatment on day 1. Subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived specimen.
- Have a performance status (PS) of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) PS.
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- No history of or current diagnosis of a 'significant autoimmune disease" or paraneoplastic autoimmune disease, i.e. myasthenia gravis, Lambert-Eaton, systemic lupus, rheumatoid arthritis. For minor 'autoimmune' disorders such as psoriasis, arthritis (not including rheumatoid arthritis), Raynaud's disease; these are allowed onto trial.
- No active hepatitis or diagnosis of human immunodeficiency virus (HIV) disease.
- No prior malignancy unless it was cured over 2 years ago; i.e. prostate cancer, or early stage (I-III) solid tumors. Patients with a prior basal skin cancer or squamous cell carcinoma of the skin or in situ cervical malignancy that have undergone curative treatment are excluded from this requirement.
- Absolute neutrophil count >= 1500/mcL (within 10 days of treatment initiation).
- Platelets >= 100000mcL (within 10 days of treatment initiation).
- Hemoglobin >= 9 g/dL or >= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment) (within 10 days of treatment initiation).
- Serum creatinine OR measured or calculated creatinine clearance (glomerular filtration rate [GFR] can also be used in place of creatinine or creatinine clearance [CrCl]) =< 1.5 X upper limit of normal (ULN) OR >= 60 mL/min for subject with creatinine levels > 1.5 X institutional ULN (within 10 days of treatment initiation). Note: Creatinine clearance should be calculated per institutional standard.
- Serum total bilirubin =< 1.5 X ULN OR direct bilirubin =< ULN for subjects with total bilirubin levels > 1.5 ULN (within 10 days of treatment initiation).
- Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 X ULN OR =< 5 X ULN for subjects with liver metastases (within 10 days of treatment initiation).
- Albumin >= 2.5 mg/dL (within 10 days of treatment initiation).
- International normalized ratio (INR) or prothrombin time (PT) activated partial thromboplastin time (aPTT) =< 1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulants =< 1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants (within 10 days of treatment initiation).
- Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
- Female subjects of childbearing potential must be willing to use an adequate method of contraception, for the course of the study through 120 days after the last dose of study medication. Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
- Male subjects of childbearing potential must agree to use an adequate method of contraception, starting with the first dose of study therapy through 120 days after the last dose of study therapy. Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
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 (i.e., =< grade 1 or at baseline) 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 (i.e., =< grade 1 or at baseline) from adverse events due to a previously administered agent. - Note: Subjects with =< grade 2 neuropathy are an exception to this criterion and may qualify for the study. - Note: If subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy.
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- Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability.
- Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
- Has a history of (non-infectious) pneumonitis that required steroids, or current 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 subject's participation for the full duration of the trial, or is not in the best interest of the subject 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 the pre-screening or 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., hepatitis B surface antigen [HBsAg] reactive) or hepatitis C (e.g., hepatitis C virus (HCV) ribonucleic acid (RNA) [qualitative] is detected).
- Has received a live vaccine within 30 days of planned start of study therapy. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines are live attenuated vaccines, and are not allowed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
M D Anderson Cancer Center
Houston, Texas, 77030
Anne S. Tsao, 713-792-6363
Anne S. Tsao
About this study
Official title: Feasibility Trial of Pembrolizumab in Unresectable Thymoma and Thymic Carcinoma
This phase I trial studies the sides effects and best dose of pembrolizumab in treating participants with thymoma or thymic cancer that cannot be removed by surgery. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the safety/tolerability of pembrolizumab in unresectable patients with thymoma or thymic carcinoma who do not have pre-existing autoimmune diseases.
SECONDARY OBJECTIVES:
- To evaluate the anti-tumor activity (response rate) of pembrolizumab in patients with unresectable thymoma or thymic carcinoma.
II. To evaluate the progression-free survival (PFS) and overall survival (OS) of pembrolizumab in patients with unresectable thymoma or thymic carcinoma.
EXPLORATORY OBJECTIVES:
- We will test archival or fresh tumor tissue for PD-L1 immunohistochemistry (IHC).
II. We will perform Guardant360 blood analysis on patients with thymoma and thymic carcinoma to determine if any particular genetic mutations correlate to toxicities or clinical outcomes to pembrolizumab.
OUTLINE: This is a dose-escalation study.
Participants receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Courses repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, participants are followed up every 6 weeks for 1 year and then every 3 months for up to 3 years.
- Conditions
- Stage III Thymoma AJCC v8, Stage IIIA Thymoma AJCC v8, Stage IIIB Thymoma AJCC v8, Stage IV Thymoma AJCC v8, Stage IVA Thymoma AJCC v8, Stage IVB Thymoma AJCC v8
- Being tested
- Pembrolizumab
- Medical subject headings
- Thymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabBiological
Also called Keytruda, Lambrolizumab, MK-3475, SCH 900475
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (pembrolizumab) | Experimental | Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Receives: Biological: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Frequency tables will be used to summarize categorical variables such toxicity type and severity.
Measured: Up to 3 years
Secondary outcomes (3)
Progression-free survival
Assessed by immune related Response Criterion (irRC) and immune-modified Response Evaluation Criteria in Solid Tumors (iRECIST). Kaplan-Meier estimates will be constructed. Confidence intervals around the median will be calculated using the Brookmeyer-Crowley method, confidence intervals around landmark time points will use the Greenwood formula for standard errors.
Measured: Up to 3 years
Overall survival assessed by iRECIST
Kaplan-Meier estimates will be constructed. Confidence intervals around the median will be calculated using the Brookmeyer-Crowley method, confidence intervals around landmark time points will use the Greenwood formula for standard errors.
Measured: Up to 3 years
Overall response rate
Assessed by RECIST 1.1 and iRECIST. Response rate and its 95% confidence interval will be calculated.
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2017
- Study start6 Dec 2017
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stage III Thymoma AJCC v8Studies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Anne S Tsao, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03295227
- Sponsor's protocol ID
- 2017-0193
- Other IDs
- NCI-2018-01029, 2017-0193
- Status last verified
- Jul 2026
- First submitted
- 25 Sep 2017
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility Trial of Pembrolizumab in Unresectable Thymoma and Thymic Carcinoma
- ClinicalTrials.gov ID
- NCT03295227
- Acronym / sponsor ID
- 2017-0193
- Conditions
- Stage III Thymoma AJCC v8, Stage IIIA Thymoma AJCC v8, Stage IIIB Thymoma AJCC v8, Stage IV Thymoma AJCC v8, Stage IVA Thymoma AJCC v8, Stage IVB Thymoma AJCC v8
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 37 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 6 Dec 2017 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 27 Sep 2017 (actual)
- 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 phase I trial studies the sides effects and best dose of pembrolizumab in treating participants with thymoma or thymic cancer that cannot be removed by surgery. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Detailed description
PRIMARY OBJECTIVE:
- To determine the safety/tolerability of pembrolizumab in unresectable patients with thymoma or thymic carcinoma who do not have pre-existing autoimmune diseases.
SECONDARY OBJECTIVES:
- To evaluate the anti-tumor activity (response rate) of pembrolizumab in patients with unresectable thymoma or thymic carcinoma.
II. To evaluate the progression-free survival (PFS) and overall survival (OS) of pembrolizumab in patients with unresectable thymoma or thymic carcinoma.
EXPLORATORY OBJECTIVES:
- We will test archival or fresh tumor tissue for PD-L1 immunohistochemistry (IHC).
II. We will perform Guardant360 blood analysis on patients with thymoma and thymic carcinoma to determine if any particular genetic mutations correlate to toxicities or clinical outcomes to pembrolizumab.
OUTLINE: This is a dose-escalation study.
Participants receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Courses repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, participants are followed up every 6 weeks for 1 year and then every 3 months for up to 3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (pembrolizumab) | Experimental | Biological: PembrolizumabParticipants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Unresectable thymoma or thymic carcinoma.
- Any line of prior therapy allowed.
- Be willing and able to provide written informed consent/assent for the trial.
- 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. Newly-obtained is defined as a specimen obtained up to 6 weeks (42 days) prior to initiation of treatment on day 1. Subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived specimen.
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 (i.e., =< grade 1 or at baseline) 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, Lambrolizumab, MK-3475, SCH 900475Given IV | Biological | Treatment (pembrolizumab) |
Primary outcome
Incidence of adverse events
Time frame: Up to 3 years
Frequency tables will be used to summarize categorical variables such toxicity type and severity.
3 secondary outcomes
Progression-free survival
Time frame: Up to 3 years
Overall survival assessed by iRECIST
Time frame: Up to 3 years
Overall response rate
Time frame: Up to 3 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 16 Jul 2026.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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.
Anne S Tsao principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Pembrolizumab in people with Stage III Thymoma AJCC v8 and Stage IIIA Thymoma AJCC v8, run by M.D. Anderson Cancer Center 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 20 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: looking for participants now, as of the sponsor's last update on 16 Jul 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Contact Anne S. Tsao (713-792-6363, astsao@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Up to 3 years). 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 16 Jul 2026.