Trial Outcomes & Findings for Pembrolizumab in Treating Patients With Stage IB-IV Mycosis Fungoides (NCT NCT03695471)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

7 months

Results posted on

2025-08-29

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Pembrolizumab) 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.
Overall Study STARTED	9
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Pembrolizumab) 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.
Overall Study Cancellation	1

Baseline Characteristics

Pembrolizumab in Treating Patients With Stage IB-IV Mycosis Fungoides

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Pembrolizumab) n=9 Participants 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.
Age, Continuous	60.3 years STANDARD_DEVIATION 12.50 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=8 Participants 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.
Proportion of Overall Cutaneous Responders at Their 9th or Final Cycle (Cutaneous Complete Response [CR], Cutaneous 90 Response [CR90] or Cutaneous Partial Response [PR])	0.25 proportion of participants

SECONDARY outcome

Timeframe: 7 months

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.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=9 Participants 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.
Incidence of Adverse Events Grade 1	5 Participants
Incidence of Adverse Events Grade 2	4 Participants

SECONDARY outcome

Timeframe: Baseline to 7 months

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.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=8 Participants 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.
Percentage Change in mSWAT Score	-5.015 Percent change from baseline Interval -97.86 to 675.19

SECONDARY outcome

Timeframe: 28 months

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.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=8 Participants 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.
Progression Free Survival	8.9 Months Interval 3.4 to Too few events, upper limit is not calculable.

SECONDARY outcome

Timeframe: 26 months

Population: All evaluable patients that achieved a response during treatment.

The distribution of duration of complete response will be estimated using the method of Kaplan-Meier.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=3 Participants 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.
Duration of Response	7.6 Months Interval 2.7 to Too few events, upper limit is not calculable.

SECONDARY outcome

Timeframe: 7 months

Population: All evaluable patients that achieved CR, CR90 or PR are included in the analysis. This includes one patient that was not a success for the primary endpoint due to progressing prior to their 9th cycle of treatment.

Median time to response is defined as the time from registration to CR, CR90 or PR.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=3 Participants 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.
Time to Response	2.04 months Interval 1.38 to 2.23

SECONDARY outcome

Timeframe: 728 days

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.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=8 Participants 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.
Median Overall Survival Time	10.6 months Interval 4.8 to Not enough events occurred; upper limit is not calculable.

Adverse Events

Treatment (Pembrolizumab)

Serious events: 1 serious events

Other events: 8 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Pembrolizumab) n=8 participants at risk 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.
Infections and infestations Fungemia	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Infections and infestations Lung infection	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Infections and infestations Sepsis	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Infections and infestations Skin infection	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Skin and subcutaneous tissue disorders Erythroderma	12.5% 1/8 • Number of events 2 • Adverse events were collected for 7 months and mortality 30 months so far

Other adverse events

Other adverse events
Measure	Treatment (Pembrolizumab) n=8 participants at risk 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.
Gastrointestinal disorders Constipation	12.5% 1/8 • Number of events 2 • Adverse events were collected for 7 months and mortality 30 months so far
Gastrointestinal disorders Diarrhea	37.5% 3/8 • Number of events 6 • Adverse events were collected for 7 months and mortality 30 months so far
Gastrointestinal disorders Nausea	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Gastrointestinal disorders Vomiting	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
General disorders Fatigue	62.5% 5/8 • Number of events 18 • Adverse events were collected for 7 months and mortality 30 months so far
Infections and infestations Skin infection	12.5% 1/8 • Number of events 1 • Adverse events were collected for 7 months and mortality 30 months so far
Investigations Creatinine increased	62.5% 5/8 • Number of events 12 • Adverse events were collected for 7 months and mortality 30 months so far
Musculoskeletal and connective tissue disorders Musculoskeletal, conn tissue - Oth spec	12.5% 1/8 • Number of events 6 • Adverse events were collected for 7 months and mortality 30 months so far
Nervous system disorders Peripheral sensory neuropathy	25.0% 2/8 • Number of events 10 • Adverse events were collected for 7 months and mortality 30 months so far
Skin and subcutaneous tissue disorders Rash maculo-papular	75.0% 6/8 • Number of events 34 • Adverse events were collected for 7 months and mortality 30 months so far
Skin and subcutaneous tissue disorders Skin and subcut tissue disord - Oth spec	25.0% 2/8 • Number of events 3 • Adverse events were collected for 7 months and mortality 30 months so far
Vascular disorders Hypertension	87.5% 7/8 • Number of events 31 • Adverse events were collected for 7 months and mortality 30 months so far

Additional Information

Jason Sluzevich

Mayo Clinic

Phone: 904-953-6402

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place