Mycosis FungoidesPhase 2NCT03695471
Pembrolizumab in Treating Patients With Stage IB-IV Mycosis Fungoides
A Phase 2 study testing Pembrolizumab in people with Mycosis Fungoides and Sezary Syndrome, run by Mayo Clinic at 1 site in United States.
Trial summary
This phase II trial studies how well pembrolizumab works in treating patients with stage IB-IV mycosis fungoides. 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mycosis Fungoides, Sezary Syndrome, Stage IB Mycosis Fungoides and Sezary Syndrome 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
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Feb 2020to 8 Oct 2024
- 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
- Age >= 18 years
- Histological confirmation of one of the following:
- Stage IIB-IV mycosis fungoides not previously treated with systemic therapy
- Stage IB/IIA mycosis fungoides with Modified Severity Weighted Assessment Tool (mSWAT) >= 20 with high risk morphologic features defined as thick plaque disease and/or follicular involvement who have failed one form of skin-directed therapy.
- Sezary syndrome patients not previously treated with systemic therapy.
- Measurable disease based on mSWAT and/or Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.
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- Be willing to provide tissue from a newly obtained core or excisional biopsy of a tumor lesion. Note: Newly-obtained is defined as a specimen obtained up to 6 weeks (42 days) prior to registration. Exception: Subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived specimen only upon agreement from the Sponsor.
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1.
- Absolute neutrophil count (ANC) >= 1,500 /mcL (obtained =< 28 days prior to registration)
- Platelet count >= 100,000/mcL (obtained =< 28 days prior to registration)
- Hemoglobin >= 9.0 g/dL or >= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment)
- Serum total bilirubin =< 1.5 X upper limit of normal (ULN) OR direct bilirubin =< ULN for subjects with total bilirubin levels > 1.5 ULN (obtained =< 28 days prior to registration)
- Aspartate transaminase (AST) and alanine transaminase (ALT) =< 2.5 X ULN OR =< 5 X ULN for subjects with liver metastases (obtained =< 28 days prior to registration)
- Albumin > 2.5 mg/dL (obtained =< 28 days prior to registration)
- Serum creatinine =< 1.5 x upper limit of normal (ULN) OR measured or calculated creatinine clearance >= 60 ml/min for subject with creatinine levels > 1.5 x institutional ULN (obtained =< 28 days prior to registration)
- Prothrombin time (PT)/international normalized ratio (INR) and partial thromboplastin time (PTT) =< 1.5 X ULN OR if patient is receiving anticoagulant therapy and PT/INR or PTT is within therapeutic range of intended use of coagulants (obtained =< 28 days prior to registration)
- Negative urine or serum pregnancy test done =< 28 days prior to registration and =< 72 hours prior to receiving the first dose of study medication, for women of childbearing potential only.
- 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 the 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 for the course of the study through 120 days after the last dose of the study medication.
- Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject.
- Provide written informed consent.
- Willing to return to enrolling institution for follow-up (during the Active Monitoring Phase of the study).
- Willing to provide tissue samples for correlative research purposes.
Who cannot join
- Any of the following because this study involves: an agent that has known genotoxic, mutagenic and teratogenic effects:
- Pregnant women
- Nursing women
- Men or women of childbearing potential who are unwilling to employ adequate contraception
- Is currently participating and receiving study therapy or have participated in a study of an investigational agent and received study therapy or used an investigational device =< 4 weeks prior to registration.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy =< 7 days prior to registration.
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- 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) =< 4 weeks prior to registration 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 =< 2 weeks prior to registration 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.
- Has a known additional malignancy that is progressing or requires active treatment. Exceptions: 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. Exceptions: subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging =< 4 weeks prior to registration 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 registration. 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). Note: 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 has 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 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 =< 30 days prior to registration.
- Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines, and are not allowed.
- Sezary syndrome patients with high blood burden requiring immediate cytoreduction.
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Florida
Jacksonville, Florida, 32224-9980
About this study
Official title: A Phase II, Open-Label, Single-Arm Trial Using KEYTRUDA (Pembrolizumab) as Initial Systemic Therapy in the Treatment of Advanced Mycosis Fungoides
This phase II trial studies how well pembrolizumab works in treating patients with stage IB-IV mycosis fungoides. 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 evaluate the antitumor activity of pembrolizumab in patients with advanced mycosis fungoides (MF) as initial systemic therapy.
SECONDARY OBJECTIVES:
- To evaluate safety of pembrolizumab in this patient population. II. To evaluate response rates of pembrolizumab in this patient population. III. To determine the progression free survival, duration of response, time to response and overall survival of pembrolizumab in this patient population.
CORRELATIVE OBJECTIVE:
- To characterize the histologic features of the anti-tumor response in patients with advanced MF before and after treatment with pembrolizumab.
OUTLINE:
Patients receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Treatment repeats every 21 days for up to 24 cycles or until complete response in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 and 90 days, and then every 3 months for up to 1 year.
- Conditions
- Mycosis Fungoides, Sezary Syndrome, Stage IB Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage II Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage IIA Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage IIB Mycosis Fungoides and Sezary Syndrome AJCC v8
- Being tested
- Pembrolizumab
- Medical subject headings
- Mycosis Fungoides, Sezary Syndrome
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 | Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 24 cycles or until complete response 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.
Proportion of Overall Cutaneous Responders at Their 9th or Final Cycle (Cutaneous Complete Response [CR], Cutaneous 90 Response [CR90] or Cutaneous Partial Response [PR])
Will be assessed by the Modified Severity Weighted Assessment Tool (mSWAT). All calculated values will use the last-observation-carried forward for any participants who withdraw, are lost to follow up, or exit the study per protocol. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients will be analyzed using Mann-Whitney U for nonparametric data and the student t-test. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner. mSWAT is calculated using body surface area (BSA) of each MF lesion (palm plus fingers of the patient ≈ 1% BSA) in each of 12 areas of the body, multiplying the sum of the BSA of each lesion type by a weighting factor (patch=1, plaque=2, and tumor =4) and generating a sum of the subtotals of each lesion subtype.
Measured: 7 months
Secondary outcomes (6)
Incidence of Adverse Events
The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. The count of participants for each maximum reported adverse event is below.
Measured: 7 months
Percentage Change in mSWAT Score
The Modified Severity-Weighted Assessment Tool (mSWAT) is used to evaluate the extent and severity of skin involvement in mycosis fungoides. It is used to standardize the assessment of skin lesions. A higher mSWAT score indicates greater extent of skin involvement. The total body surface area (BSA) affected by each type of lesion (patches, plaques, and tumors) is estimated, and each lesion type is assigned a weighting factor: patches *1, plaques *2, and tumors *4. The BSA for each lesion type is multiplied by its corresponding weighting factor, and then the weighted values are summed to produce the final mSWAT score. mSWAT is calculated using body surface area (BSA) of each MF lesion (palm plus fingers of the patient ≈ 1% BSA) in each of 12 areas of the body, multiplying the sum of the BSA of each lesion type by a weighting factor (patch=1, plaque=2, and tumor =4) and generating a sum of the subtotals of each lesion subtype.
Measured: Baseline to 7 months
Progression Free Survival
The distribution of progression-free survival will be estimated using the method of Kaplan-Meier. In addition, the progression-free survival rate at 5 years after registration will be reported.
Measured: 28 months
Duration of Response
The distribution of duration of complete response will be estimated using the method of Kaplan-Meier.
Measured: 26 months
Time to Response
Median time to response is defined as the time from registration to CR, CR90 or PR.
Measured: 7 months
Median Overall Survival Time
The distribution of survival time will be estimated using the method of Kaplan-Meier. It's defined as the time from a patients registration until death or lost to followup.
Measured: 728 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2018
- Study start4 Feb 2020
- Primary completion21 Jun 2023
- Study completion8 Oct 2024
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 Mycosis FungoidesStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jason C. Sluzevich, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03695471
- Sponsor's protocol ID
- MC1788
- Other IDs
- NCI-2018-02023, 17-010349
- Status last verified
- Dec 2024
- First submitted
- 2 Oct 2018
- Last updated
- 29 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-Label, Single-Arm Trial Using KEYTRUDA (Pembrolizumab) as Initial Systemic Therapy in the Treatment of Advanced Mycosis Fungoides
- ClinicalTrials.gov ID
- NCT03695471
- Acronym / sponsor ID
- MC1788
- Conditions
- Mycosis Fungoides, Sezary Syndrome, Stage IB Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage II Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage IIA Mycosis Fungoides and Sezary Syndrome AJCC v8, Stage IIB Mycosis Fungoides and Sezary Syndrome AJCC v8
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 4 Feb 2020 (actual)
- Primary completion
- 21 Jun 2023 (actual)
- Study completion
- 8 Oct 2024 (actual)
- First posted
- 4 Oct 2018 (actual)
- Results
- Posted 11 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well pembrolizumab works in treating patients with stage IB-IV mycosis fungoides. 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 evaluate the antitumor activity of pembrolizumab in patients with advanced mycosis fungoides (MF) as initial systemic therapy.
SECONDARY OBJECTIVES:
- To evaluate safety of pembrolizumab in this patient population. II. To evaluate response rates of pembrolizumab in this patient population. III. To determine the progression free survival, duration of response, time to response and overall survival of pembrolizumab in this patient population.
CORRELATIVE OBJECTIVE:
- To characterize the histologic features of the anti-tumor response in patients with advanced MF before and after treatment with pembrolizumab.
OUTLINE:
Patients receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Treatment repeats every 21 days for up to 24 cycles or until complete response in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 and 90 days, and then every 3 months for up to 1 year.
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: PembrolizumabPatients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 24 cycles or until complete response 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 (24 listed)
- Age >= 18 years
- Histological confirmation of one of the following:
- Measurable disease based on mSWAT and/or 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. Note: Newly-obtained is defined as a specimen obtained up to 6 weeks (42 days) prior to registration. Exception: Subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived specimen only upon agreement from the Sponsor.
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1.
Key exclusion criteria (26 listed)
- Any of the following because this study involves: an agent that has known genotoxic, mutagenic and teratogenic effects:
- Is currently participating and receiving study therapy or have participated in a study of an investigational agent and received study therapy or used an investigational device =< 4 weeks prior to registration.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy =< 7 days prior to registration.
- Has a known history of active TB (Bacillus tuberculosis).
- Hypersensitivity to pembrolizumab or any of its excipients.
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
Proportion of Overall Cutaneous Responders at Their 9th or Final Cycle (Cutaneous Complete Response [CR], Cutaneous 90 Response [CR90] or Cutaneous Partial Response [PR])
Time frame: 7 months
Will be assessed by the Modified Severity Weighted Assessment Tool (mSWAT). All calculated values will use the last-observation-carried forward for any participants who withdraw, are lost to follow up, or exit the study per protocol. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients will be analyzed using Mann-Whitney U for nonparametric data and the student t-test. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner. mSWAT is calculated using body surface area (BSA) of each MF lesion (palm plus fingers of the patient ≈ 1% BSA) in each of 12 areas of the body, multiplying the sum of the BSA of each lesion type by a weighting factor (patch=1, plaque=2, and tumor =4) and generating a sum of the subtotals of each lesion subtype.
6 secondary outcomes
Incidence of Adverse Events
Time frame: 7 months
Percentage Change in mSWAT Score
Time frame: Baseline to 7 months
Progression Free Survival
Time frame: 28 months
Duration of Response
Time frame: 26 months
Time to Response
Time frame: 7 months
Median Overall Survival Time
Time frame: 728 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
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Golidocitinib in Patients With Mycosis Fungoides/Sézary Syndrome and T-Cell Large Granular Lymphocytic Leukemia
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RecruitingObservational study
A Prospective, US-based Study Assessing Mogamulizumab-associated Rash in Patients Diagnosed With Mycosis Fungoides or Sezary Syndrome and Treated With Standard of Care Mogamulizumab
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Duvelisib and Nivolumab for the Treatment of Stage IIB-IVB Mycosis Fungoides and Sezary Syndrome
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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.
Jason C. Sluzevich, M.D. principal investigator
Mayo Clinic
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 Mycosis Fungoides and Sezary Syndrome, run by Mayo Clinic 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 24 inclusion and 26 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: finished, as of the sponsor's last update on 29 Aug 2025. 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 11 Aug 2025). 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?
Proportion of Overall Cutaneous Responders at Their 9th or Final Cycle (Cutaneous Complete Response [CR], Cutaneous 90 Response [CR90] or Cutaneous Partial Response [PR]) (time frame: 7 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 29 Aug 2025.