Trial Outcomes & Findings for Pembrolizumab in Treating Patients With Relapsed or Refractory Stage IB-IVB Mycosis Fungoides or Sezary Syndrome (NCT NCT02243579)
NCT ID: NCT02243579
Last Updated: 2019-09-20
Results Overview
A generalized linear model for the objective response rate used a binominal error distribution. The model included as covariates all available baseline predictors of the missing outcomes.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
Up to 3.2 years
Results posted on
2019-09-20
Participant Flow
Participant milestones
| Measure |
Treatment (Pembrolizumab)
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
20
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pembrolizumab in Treating Patients With Relapsed or Refractory Stage IB-IVB Mycosis Fungoides or Sezary Syndrome
Baseline characteristics by cohort
| Measure |
Treatment (Pembrolizumab)
n=24 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 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
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3.2 yearsA generalized linear model for the objective response rate used a binominal error distribution. The model included as covariates all available baseline predictors of the missing outcomes.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=24 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Objective Response Rate (ORR), Defined as a Confirmed Partial Response (PR) or Complete Response (CR) Using Global Assessment Standard Response Criteria for Mycosis Fungoides and Sezary Syndrome
Confirmed Complete Response
|
2 Participants
|
|
Objective Response Rate (ORR), Defined as a Confirmed Partial Response (PR) or Complete Response (CR) Using Global Assessment Standard Response Criteria for Mycosis Fungoides and Sezary Syndrome
Confirmed Partial Response
|
7 Participants
|
|
Objective Response Rate (ORR), Defined as a Confirmed Partial Response (PR) or Complete Response (CR) Using Global Assessment Standard Response Criteria for Mycosis Fungoides and Sezary Syndrome
Stable Disease
|
9 Participants
|
|
Objective Response Rate (ORR), Defined as a Confirmed Partial Response (PR) or Complete Response (CR) Using Global Assessment Standard Response Criteria for Mycosis Fungoides and Sezary Syndrome
Progressive Disease
|
6 Participants
|
SECONDARY outcome
Timeframe: The time interval between the date of first response (CR/PR) and the date of progression as assessed by standard Mycosis Fungoides and Sezary Syndrome response criteria, assessed at 26 and 52 weeksWas estimated using the Kaplan-Meier method (Duration of Response Probability).
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=9 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Duration of Response
26 weeks
|
1.0 Duration of Response Probability
|
|
Duration of Response
52 weeks
|
0.8571 Duration of Response Probability
|
SECONDARY outcome
Timeframe: The time from allocation to the first documented disease progression or death due to any cause, whichever occurs first, assessed at 26 and 52 weeksWas estimated using the Kaplan-Meier method (Progression Free Survival Probability).
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=24 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Progression Free Survival (PFS)
26 weeks
|
0.7380 Progression Free Survival Probability
|
|
Progression Free Survival (PFS)
52 weeks
|
0.6457 Progression Free Survival Probability
|
SECONDARY outcome
Timeframe: The time from randomization to death due to any cause, assessed at 52, 104 and 156 weeksWas estimated using the Kaplan-Meier method (Overall Survival Probability).
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=24 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Overall Survival (OS)
52 weeks
|
0.95 Overall Survival Probability
|
|
Overall Survival (OS)
104 weeks
|
0.7917 Overall Survival Probability
|
|
Overall Survival (OS)
156 weeks
|
0.5429 Overall Survival Probability
|
SECONDARY outcome
Timeframe: Up to 4 yearsPopulation: Patients with drug-related toxicities included in the analysis
Summary statistics (mean and SD) for time to onset of first drug-related toxicity was provided.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=22 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Time to Onset of First Drug-related Toxicity
|
64.5 Days
Standard Deviation 121.3
|
SECONDARY outcome
Timeframe: Up to 4 yearsAdverse events were summarized as counts and frequencies by toxicity grade.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=24 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Incidence of Adverse Events Graded Using the Common Terminology Criteria for Adverse Events Version 5.0
Maximum Toxicity Grade 1
|
2 Participants
|
|
Incidence of Adverse Events Graded Using the Common Terminology Criteria for Adverse Events Version 5.0
Maximum Toxicity Grade 2
|
7 Participants
|
|
Incidence of Adverse Events Graded Using the Common Terminology Criteria for Adverse Events Version 5.0
Maximum Toxicity Grade 3
|
9 Participants
|
|
Incidence of Adverse Events Graded Using the Common Terminology Criteria for Adverse Events Version 5.0
Maximum Toxicity Grade 4
|
6 Participants
|
Adverse Events
Treatment (Pembrolizumab)
Serious events: 11 serious events
Other events: 24 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Treatment (Pembrolizumab)
n=24 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Cardiac disorders
Atrial Fibrillation
|
4.2%
1/24 • Up to 4 years
|
|
Cardiac disorders
Heart Failure
|
4.2%
1/24 • Up to 4 years
|
|
Eye disorders
Corneal Ulcer
|
4.2%
1/24 • Up to 4 years
|
|
Eye disorders
Periorbital Edema
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Colitis
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Colonic obstruction
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Duodenitis
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Edema face
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Fatigue
|
4.2%
1/24 • Up to 4 years
|
|
Immune system disorders
Anaphylaxis
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Lung Infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Sepsis
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Urinary Tract Infection
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
Alanine Aminotransferase increased
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Aspartate Aminotransferase increased
|
4.2%
1/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.2%
1/24 • Up to 4 years
|
|
Psychiatric disorders
Confusion
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneuminitis
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Edema
|
4.2%
1/24 • Up to 4 years
|
|
Vascular disorders
Hypertension
|
4.2%
1/24 • Up to 4 years
|
Other adverse events
| Measure |
Treatment (Pembrolizumab)
n=24 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for up to 2 years (6 months for patients achieving CR) in the absence of disease progression or unacceptable toxicity.
Laboratory Biomarker Analysis: Correlative studies
Pembrolizumab: Given IV
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
29.2%
7/24 • Up to 4 years
|
|
Blood and lymphatic system disorders
Low Hemoglobin
|
4.2%
1/24 • Up to 4 years
|
|
Ear and labyrinth disorders
Vertigo
|
8.3%
2/24 • Up to 4 years
|
|
Eye disorders
Blurred Vision
|
4.2%
1/24 • Up to 4 years
|
|
Eye disorders
Keratitis
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Abdominal Pain
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Colonic hemorrhage
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Constipation
|
4.2%
1/24 • Up to 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
12.5%
3/24 • Up to 4 years
|
|
Gastrointestinal disorders
Nausia
|
8.3%
2/24 • Up to 4 years
|
|
Gastrointestinal disorders
Vomiting
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Chills
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Edema face
|
8.3%
2/24 • Up to 4 years
|
|
General disorders
Edema limbs
|
16.7%
4/24 • Up to 4 years
|
|
General disorders
Edema trunk
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Fatigue
|
29.2%
7/24 • Up to 4 years
|
|
General disorders
Fever
|
8.3%
2/24 • Up to 4 years
|
|
General disorders
Weakness
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Weakness (grade decrease)
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Firm tender lump on his left lateral thigh, no trauma to the area
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Generalized edema
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Malaise
|
4.2%
1/24 • Up to 4 years
|
|
General disorders
Pain
|
4.2%
1/24 • Up to 4 years
|
|
Immune system disorders
Inflammation/Flare reaction
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Conjunctivitis
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Esophageal infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Eye infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Infection S/P scheduled Mohs surgery
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Right lower leg infection related to approved radiation and wound care
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Mucosal infection (thrush)
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Mucosal infection - thrush
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Upper eyelid infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
otitis externa
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
rash pustular
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Skin infection
|
8.3%
2/24 • Up to 4 years
|
|
Infections and infestations
Soft tissue infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Upper respiratory infection
|
4.2%
1/24 • Up to 4 years
|
|
Infections and infestations
Urinary tract infection
|
4.2%
1/24 • Up to 4 years
|
|
Injury, poisoning and procedural complications
Fall
|
4.2%
1/24 • Up to 4 years
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
Alanine aminotransferase increased
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
Alkaline phosphatase inceased
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Aspartate aminotransferase increased
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
Blood bilirubin increased
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
Cardiac troponin T increased
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Creatinine increased
|
16.7%
4/24 • Up to 4 years
|
|
Investigations
Investigations
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Lymphocyte count decreased
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Neutrophil count decreased
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Weight gain
|
4.2%
1/24 • Up to 4 years
|
|
Investigations
Weight loss
|
8.3%
2/24 • Up to 4 years
|
|
Investigations
white blood cell decreased
|
4.2%
1/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
25.0%
6/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
4.2%
1/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
20.8%
5/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
8.3%
2/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
20.8%
5/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
4.2%
1/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
8.3%
2/24 • Up to 4 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
3/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
2/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Herniated disc
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
neck pain
|
4.2%
1/24 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.5%
3/24 • Up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
BCC right leg
|
4.2%
1/24 • Up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
SCC forehead
|
4.2%
1/24 • Up to 4 years
|
|
Nervous system disorders
Ataxia
|
4.2%
1/24 • Up to 4 years
|
|
Nervous system disorders
Syncope
|
4.2%
1/24 • Up to 4 years
|
|
Nervous system disorders
Tremor
|
4.2%
1/24 • Up to 4 years
|
|
Psychiatric disorders
Anxiety
|
4.2%
1/24 • Up to 4 years
|
|
Psychiatric disorders
Confusion
|
12.5%
3/24 • Up to 4 years
|
|
Psychiatric disorders
Insomnia
|
8.3%
2/24 • Up to 4 years
|
|
Renal and urinary disorders
Nephrolithiasis
|
4.2%
1/24 • Up to 4 years
|
|
Renal and urinary disorders
Acute kidney injury
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
4/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.2%
1/24 • Up to 4 years
|
|
Reproductive system and breast disorders
Horseness
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.3%
2/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal drip
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Bilateral pulmonary infiltrates
|
4.2%
1/24 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Sinus inflammation
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
12.5%
3/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Erythroderma
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.8%
5/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin eruption/flare, rash
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
immune mediated flare reaction on the face
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
pustular rash
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Mild skin peeling
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin flare
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Excess skin peeling
|
4.2%
1/24 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
4.2%
1/24 • Up to 4 years
|
|
Surgical and medical procedures
Scheduled Mohs surgery
|
4.2%
1/24 • Up to 4 years
|
|
Vascular disorders
Hot flashes
|
4.2%
1/24 • Up to 4 years
|
|
Vascular disorders
Hypertension
|
4.2%
1/24 • Up to 4 years
|
|
Vascular disorders
Hypotension
|
4.2%
1/24 • Up to 4 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60