Trial Outcomes & Findings for Erlotinib Prevention of Oral Cancer (EPOC) (NCT NCT00402779)
NCT ID: NCT00402779
Last Updated: 2020-04-21
Results Overview
Cancer-free survival defined as time from randomization to the development of histologically confirmed oral cancer.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
303 participants
Primary outcome timeframe
3 years
Results posted on
2020-04-21
Participant Flow
Patients seen at the following sites exhibiting oral leukoplakia we approached and enrolled if interested. These include MD Anderson Cancer Center, Memorial Sloan-Kettering, Emory University,the University of Chicago, and the University of Maryland.
Patients with leukoplakia were screened and, once eligibility was confirmed, patients willing to proceed were randomized to erlotinib or placebo in a double-blinded manner.
Participant milestones
| Measure |
Erlotinib
Erlotinib 150 mg PO daily x 12 months
|
Placebo
Placebo PO daily x 12 months
|
|---|---|---|
|
Overall Study
STARTED
|
152
|
151
|
|
Overall Study
COMPLETED
|
75
|
75
|
|
Overall Study
NOT COMPLETED
|
77
|
76
|
Reasons for withdrawal
| Measure |
Erlotinib
Erlotinib 150 mg PO daily x 12 months
|
Placebo
Placebo PO daily x 12 months
|
|---|---|---|
|
Overall Study
Screen Failure
|
77
|
76
|
Baseline Characteristics
Erlotinib Prevention of Oral Cancer (EPOC)
Baseline characteristics by cohort
| Measure |
Erlotinib
n=75 Participants
Erlotinib 150 mg PO daily x 12 months
|
Placebo
n=75 Participants
Placebo PO daily x 12 months
|
Total
n=150 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
21-30 years
|
3 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
|
Age, Customized
31-40 years
|
6 Participants
n=39 Participants
|
9 Participants
n=41 Participants
|
15 Participants
n=35 Participants
|
|
Age, Customized
41-50 years
|
14 Participants
n=39 Participants
|
13 Participants
n=41 Participants
|
27 Participants
n=35 Participants
|
|
Age, Customized
51-60 years
|
21 Participants
n=39 Participants
|
25 Participants
n=41 Participants
|
46 Participants
n=35 Participants
|
|
Age, Customized
61-70 years
|
20 Participants
n=39 Participants
|
22 Participants
n=41 Participants
|
42 Participants
n=35 Participants
|
|
Age, Customized
71-80 years
|
10 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
14 Participants
n=35 Participants
|
|
Age, Customized
81-90 years
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Sex: Female, Male
Female
|
32 Participants
n=39 Participants
|
33 Participants
n=41 Participants
|
65 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
43 Participants
n=39 Participants
|
42 Participants
n=41 Participants
|
85 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
70 Participants
n=39 Participants
|
70 Participants
n=41 Participants
|
140 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
3 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
68 Participants
n=39 Participants
|
67 Participants
n=41 Participants
|
135 Participants
n=35 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=39 Participants
|
6 Participants
n=41 Participants
|
9 Participants
n=35 Participants
|
|
Region of Enrollment
United States
|
73 participants
n=39 Participants
|
75 participants
n=41 Participants
|
148 participants
n=35 Participants
|
|
Region of Enrollment
Europe
|
2 participants
n=39 Participants
|
0 participants
n=41 Participants
|
2 participants
n=35 Participants
|
PRIMARY outcome
Timeframe: 3 yearsCancer-free survival defined as time from randomization to the development of histologically confirmed oral cancer.
Outcome measures
| Measure |
Erlotinib
n=75 Participants
Erlotinib 150 mg PO daily x 12 months
|
Placebo
n=75 Participants
Placebo PO daily x 12 months
|
|---|---|---|
|
Oral Cancer-free Survival in Participants Receiving Erlotinib as Compared With the Control Arm or Placebo Group.
|
55 Participants
|
18 Participants
|
Adverse Events
Erlotinib
Serious events: 10 serious events
Other events: 68 other events
Deaths: 6 deaths
Placebo
Serious events: 4 serious events
Other events: 32 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
Erlotinib
n=75 participants at risk
Erlotinib 150 mg PO daily x 12 months
|
Placebo
n=75 participants at risk
Placebo PO daily x 12 months
|
|---|---|---|
|
Cardiac disorders
Cardiac ischemia/infarction
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Rash Acneiform
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Infections and infestations
Infection
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
2.7%
2/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Dehydration
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Nervous system disorders
Speech Impairment
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Nervous system disorders
Confusion
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Nervous system disorders
Dizzines
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Nervous system disorders
Blurred Vision
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain
|
2.7%
2/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Infections and infestations
Pneumonia
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Reproductive system and breast disorders
Altered Sexual Function
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
Other adverse events
| Measure |
Erlotinib
n=75 participants at risk
Erlotinib 150 mg PO daily x 12 months
|
Placebo
n=75 participants at risk
Placebo PO daily x 12 months
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
18.7%
14/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
26.7%
20/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Blood and lymphatic system disorders
Decreased Leukocytes
|
13.3%
10/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
General disorders
Fatigue
|
61.3%
46/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
28.0%
21/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
General disorders
Fever
|
12.0%
9/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
General disorders
Insomnia
|
12.0%
9/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Weight loss
|
6.7%
5/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
18.7%
14/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Derm Other
|
16.0%
12/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
40.0%
30/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
17.3%
13/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
65.3%
49/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
20.0%
15/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.7%
38/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
25.3%
19/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Rash-acneiform
|
86.7%
65/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
16.0%
12/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
90.7%
68/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
24.0%
18/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Distension
|
6.7%
5/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Dry mouth
|
12.0%
9/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Dysphagia
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Flatulance
|
6.7%
5/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Dyspepsia
|
18.7%
14/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Mucositis
|
20.0%
15/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
9.3%
7/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Epistaxis
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Rectal bleeding
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
General disorders
Edema/head and neck
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Elevated Alk Phos
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Elevated ALT
|
13.3%
10/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
17.3%
13/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Elevated Creatinine
|
12.0%
9/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
9.3%
7/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Elevated bilirubin
|
21.3%
16/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
24.0%
18/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Elevated AST
|
32.0%
24/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Hyperkalemia
|
14.7%
11/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
1.3%
1/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Hypomagnesemia
|
22.7%
17/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Investigations
Hyponatremia
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Cardiac disorders
Dizziness
|
12.0%
9/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Psychiatric disorders
Depression
|
9.3%
7/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Eye disorders
Dry Eye
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Pain
|
25.3%
19/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Pain
|
33.3%
25/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
32.0%
24/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
6.7%
5/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.7%
14/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Cardiac disorders
Hypertension
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
8.0%
6/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
16.0%
12/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
Infections and infestations
Sinus Infection
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
5.3%
4/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
|
General disorders
Headache
|
0.00%
0/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
20.0%
15/75 • From drug start through 30 days after drug discontinuation, an average of 3 years
|
Additional Information
Dr. John V. Heymach, PHD/Chair, Thoracic-Head & Neck Med Onc
UT MD Anderson Cancer Center
Phone: 713-792-6363
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place