Trial Outcomes & Findings for Pembrolizumab in Treating Patients With High Risk Oral Intraepithelial Neoplasia (NCT NCT02882282)
NCT ID: NCT02882282
Last Updated: 2025-10-23
Results Overview
To determine oral cancer-free survival and overall survival of patients with high risk oral IEN treated with Pembrolizumab versus observation
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
51.2 months
Results posted on
2025-10-23
Participant Flow
Participant milestones
| Measure |
Arm B (Pembrolizumab).
Pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 4 courses until disease progression or unacceptable toxicity
|
Arm A (Control)
Observation
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
7
|
|
Overall Study
COMPLETED
|
7
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Arm B (Pembrolizumab).
Pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 4 courses until disease progression or unacceptable toxicity
|
Arm A (Control)
Observation
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
Pembrolizumab in Treating Patients With High Risk Oral Intraepithelial Neoplasia
Baseline characteristics by cohort
| Measure |
Arm A (Control)
n=7 Participants
Observation
|
Arm B (Pembrolizumab)
n=8 Participants
Pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 4 courses until disease progression or unacceptable toxicity
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
8 participants
n=7 Participants
|
15 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 51.2 monthsPopulation: Intent-to-treat population
To determine oral cancer-free survival and overall survival of patients with high risk oral IEN treated with Pembrolizumab versus observation
Outcome measures
| Measure |
Arm A (Control)
n=7 Participants
Observation
|
Arm B (Pembrolizumab )
n=8 Participants
Pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 4 courses until disease progression or unacceptable toxicity
|
|---|---|---|
|
Oral Cancer-free Survival
|
NA Months
Not reached
|
6.7 Months
Interval 3.6 to 40.2
|
SECONDARY outcome
Timeframe: Overall survival was defined as from the randomization to death of all causes, whichever occurred first or last follow up.Population: Intent-to-treat population
To determine overall survival of patients with high risk oral IEN treated with pembrolizumab versus observation. The distribtuion of overall survival was estimated using Kaplan-Meier method.
Outcome measures
| Measure |
Arm A (Control)
n=7 Participants
Observation
|
Arm B (Pembrolizumab )
n=8 Participants
Pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for up to 4 courses until disease progression or unacceptable toxicity
|
|---|---|---|
|
Overall Survival
|
NA months
Not reached
|
NA months
Not reached
|
Adverse Events
Arm A
Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths
Arm B
Serious events: 0 serious events
Other events: 6 other events
Deaths: 7 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Arm A
n=7 participants at risk
Observation (Control)
|
Arm B
n=8 participants at risk
Pembrolizumab 4 doses (experimental)
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/7 • Approximately 4 years, 4 months
|
50.0%
4/8 • Approximately 4 years, 4 months
|
|
General disorders
Fatigue
|
0.00%
0/7 • Approximately 4 years, 4 months
|
37.5%
3/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Mucositis Oral
|
0.00%
0/7 • Approximately 4 years, 4 months
|
25.0%
2/8 • Approximately 4 years, 4 months
|
|
General disorders
Hypothyroidism
|
0.00%
0/7 • Approximately 4 years, 4 months
|
25.0%
2/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/7 • Approximately 4 years, 4 months
|
25.0%
2/8 • Approximately 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/7 • Approximately 4 years, 4 months
|
25.0%
2/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Non-Cardiac chest pain
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Infections and infestations
Sepsis
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Allergic Rhinitis
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Cardiac disorders
Chest Pain - Cardiac
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
General disorders
Ear Pain
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
General disorders
Flu Like Symptoms
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Nervous system disorders
Headache
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Vascular disorders
Hot flashes
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Joint Pain
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Oral secretion
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
Peg Placement
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Infections and infestations
Rash pustular
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Gastrointestinal disorders
vomiting
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
|
Investigations
White blood cell decreased
|
0.00%
0/7 • Approximately 4 years, 4 months
|
12.5%
1/8 • Approximately 4 years, 4 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place