Trial Outcomes & Findings for Pembrolizumab in Treating Patients With Advanced Uveal Melanoma (NCT NCT02359851)
NCT ID: NCT02359851
Last Updated: 2019-09-18
Results Overview
Of note, response rates by RECIST 1.1 criteria will also be assessed although irRC will be used for the primary endpoint. The percentage of complete or partial response is calculated and 95% confidence interval is estimated using Wilson method.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
Up to 3 years
Results posted on
2019-09-18
Participant Flow
The recruitment period for this trial was May 2015 to January 2017. The trial was conducted at Vanderbilt-Ingram Cancer Center in Nashville, Tennessee and University of Chicago in Chicago, Illinois This trial closed due to slow accrual.
Participant milestones
| Measure |
Treatment (Pembrolizumab)
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Study
STARTED
|
5
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Treatment (Pembrolizumab)
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Disease progression
|
1
|
Baseline Characteristics
Pembrolizumab in Treating Patients With Advanced Uveal Melanoma
Baseline characteristics by cohort
| Measure |
Treatment (Pembrolizumab)
n=5 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
61 Years
STANDARD_DEVIATION 16.7332 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 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
|
0 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
|
|
Region of Enrollment
United States
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3 yearsPopulation: Patients received pembrolizumab treatment and evaluated for response.
Of note, response rates by RECIST 1.1 criteria will also be assessed although irRC will be used for the primary endpoint. The percentage of complete or partial response is calculated and 95% confidence interval is estimated using Wilson method.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=4 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Objective Response Rate (ORR), Defined as the Percentage of Patients Achieving a Confirmed Complete or Partial Response, as Defined by Immune-related Response Criteria (irRC)
|
25 % of patients achieving response
Interval 1.3 to 69.9
|
SECONDARY outcome
Timeframe: Time from the first dose of pembrolizumab to progression, assessed up to 3 years.Population: Patients received treatment.
PFS will be summarized using the method of Kaplan and Meier. Median PFS time with 95% confidence intervals will be reported. Using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), progression will be defined as ≥ 20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest diameter recorded or the appearance of one or more new lesions, and/or appearance of one or more new lesions and/or unequivocal progression of existing nontarget lesions.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=5 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Progression-Free Survival (PFS) Defined as Time From Treatment to Progression Defined by RECIST1.1 Criteria or Death.
|
36.0 months
Insufficient patient data.
|
SECONDARY outcome
Timeframe: Interval between the first dose of pembrolizumab and death for any reason, assessed up to 3 yearsPopulation: Patients received pembrolizumab treatment.
The Kaplan-Meier method will be used to estimate the overall survival. Median survival and its 95% confidence interval will be estimated and reported.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=5 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Survival (OS) Defined as Number of Patients Surviving up to 3 Years.
Survived
|
3 Participants
|
|
Overall Survival (OS) Defined as Number of Patients Surviving up to 3 Years.
Died
|
2 Participants
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Only 1 participant had a RECIST-defined response, which is ongoing and lasting 23.5 months.
If sample size permits, response duration will be summarized descriptively using Kaplan-Meier medians and quartiles. Only the subset of patients who show a complete response or partial response will be included in this analysis.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=1 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Response Duration
|
23.5 months
|
SECONDARY outcome
Timeframe: Up to 30 days after the last dose of trial treatmentCount of patients according to the worst-grade toxicity experienced by each, where worst-grade toxicity is per NCI common toxicity criteria: grade 1, mild; grade 2, moderate; grade 3, severe; grade 4, life-threatening; grade 5, death. This endpoint will be reported descriptively without formal statistical analysis.
Outcome measures
| Measure |
Treatment (Pembrolizumab)
n=4 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Number of Patients With Each Worst-Grade Toxicity. Incidence of Adverse Events, Using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Number of participants with worst-grade toxicity 1
|
1 Participants
|
|
Number of Patients With Each Worst-Grade Toxicity. Incidence of Adverse Events, Using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Number of participants with worst-grade toxicity 2
|
1 Participants
|
|
Number of Patients With Each Worst-Grade Toxicity. Incidence of Adverse Events, Using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Number of participants with worst-grade toxicity 3
|
0 Participants
|
|
Number of Patients With Each Worst-Grade Toxicity. Incidence of Adverse Events, Using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Number of participants with worst-grade toxicity 4
|
1 Participants
|
|
Number of Patients With Each Worst-Grade Toxicity. Incidence of Adverse Events, Using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Number of participants with worst-grade toxicity 5
|
1 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to 2 yearsDifferences in PFS and OS between subgroups will be assessed by the stratified logrank test. Differences in ORR between subgroups will be assessed by the Pearson chi-square test. No adjustments will be made for multiple comparisons.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to 2 yearsDifferences in PFS and OS between subgroups will be assessed by the stratified logrank test. Differences in ORR between subgroups will be assessed by the Pearson chi-square test. No adjustments will be made for multiple comparisons.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to 2 yearsEach relevant clinical characteristic will be recorded by the use of tables and/or graphs. The number and percentage of patients treated, and the primary reason for discontinuation will be displayed. Demographic variables (such as age) and baseline characteristics will be summarized by descriptive statistics. Compliance with pembrolizumab administration will be measured by patients: 1) receiving unscheduled study agent infusions/injections; 2) missing an infusion/injection. Numbers and percentages of patients and infusion/injection visits with any deviation in these measures will be reported.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (Pembrolizumab)
Serious events: 3 serious events
Other events: 4 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Treatment (Pembrolizumab)
n=5 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Investigations
Increased Creatinine
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Investigations
INR Increased
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Renal and urinary disorders
Acute Kidney Inury
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Lower GI Hemorrhage
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Investigations
Blood bilirubin increased
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
hyperglycemia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Small bowel obstruction
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Perforate diverticulitis
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Nervous system disorders
Encephalopathy
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplassms
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
Other adverse events
| Measure |
Treatment (Pembrolizumab)
n=5 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity.
Pembrolizumab: Given IV
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
General disorders
Fatigue
|
60.0%
3/5 • Number of events 5 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
General disorders
Limb edema
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
General disorders
Pain
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Nausea
|
40.0%
2/5 • Number of events 3 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Bloating
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Constipation
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Dry mouth
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Ileus
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Investigations
Aspartate aminotransferase increased
|
40.0%
2/5 • Number of events 7 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Investigations
Alanine aminotransferase increased
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Investigations
Alkaline phosphatase increased
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hyponatremia
|
40.0%
2/5 • Number of events 5 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
40.0%
2/5 • Number of events 4 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
20.0%
1/5 • Number of events 4 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Anorexia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Blood and lymphatic system disorders
Anemia
|
40.0%
2/5 • Number of events 5 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Endocrine disorders
Hypothyroidism
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Eye disorders
Dry Eye
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Eye disorders
Eye pain
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Eye disorders
Retinal vascular disorder
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Infections and infestations
Upper respiratory infection
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Infections and infestations
Urinary tract infection
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Nervous system disorders
Dysgeusia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Injury, poisoning and procedural complications
Bruising
|
20.0%
1/5 • Number of events 2 • From the time participants signed consent form through 30 days following cessation of treatment .
|
|
Psychiatric disorders
Insomnia
|
20.0%
1/5 • Number of events 1 • From the time participants signed consent form through 30 days following cessation of treatment .
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place