Trial Outcomes & Findings for Everolimus Versus Placebo in Head and Neck Cancer (NCT NCT01111058)
NCT ID: NCT01111058
Last Updated: 2020-04-07
Results Overview
Time to disease progression or death from any cause--2 year rate
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
52 participants
Primary outcome timeframe
2 years
Results posted on
2020-04-07
Participant Flow
Participant milestones
| Measure |
Everolimus (RAD001)
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
24
|
|
Overall Study
COMPLETED
|
28
|
24
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Everolimus Versus Placebo in Head and Neck Cancer
Baseline characteristics by cohort
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
Total
n=52 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Region of Enrollment
United States
|
28 participants
n=39 Participants
|
24 participants
n=41 Participants
|
52 participants
n=35 Participants
|
|
Age, Continuous
|
58.0 years
n=39 Participants
|
57.4 years
n=41 Participants
|
57.7 years
n=35 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
9 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=39 Participants
|
20 Participants
n=41 Participants
|
43 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
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
6 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=39 Participants
|
20 Participants
n=41 Participants
|
45 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
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
PRIMARY outcome
Timeframe: 2 yearsTime to disease progression or death from any cause--2 year rate
Outcome measures
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
2 Year Progression Free Survival Rate
|
56.1 percentage of patients
Interval 31.1 to 81.2
|
55.9 percentage of patients
Interval 34.1 to 77.8
|
SECONDARY outcome
Timeframe: 4 yearsAdverse event rate, any type, any grade regardless of attribution
Outcome measures
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Number of Participants With Toxicity
|
25 Participants
|
16 Participants
|
SECONDARY outcome
Timeframe: 4 yearsNumber of patients with local-regional progression
Outcome measures
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Site of Progression: Local-regional
|
2 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: 4 yearsNumber of patients with distant progression
Outcome measures
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Site of Progression: Distant
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 4 yearsNumber of patients with unknown site of progression
Outcome measures
| Measure |
Everolimus (RAD001)
n=28 Participants
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 Participants
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Site of Progression: Unknown
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 4 yearsPopulation: Data not systematically recorded.
Number of patients with second primary tumor
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: BaselinePopulation: Data were not collected.
mTOR positive in tumor tissue
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 4 yearsPopulation: Data were not collected.
mTOR positive in tumor tissue
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 4 yearsPopulation: Data were not collected.
Differential effect of PTEN status on progression-free survival between the two arms
Outcome measures
Outcome data not reported
Adverse Events
Everolimus (RAD001)
Serious events: 3 serious events
Other events: 25 other events
Deaths: 4 deaths
Placebo
Serious events: 5 serious events
Other events: 14 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Everolimus (RAD001)
n=28 participants at risk
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 participants at risk
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Infections and infestations
Pharyngitis
|
3.6%
1/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Infections and infestations
Skin infection
|
3.6%
1/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Vascular disorders
Thromboembolic event
|
3.6%
1/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
General disorders
Dysphagia
|
0.00%
0/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Vascular disorders
Hypertension
|
0.00%
0/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukemia secondary to oncology chemotherapy
|
0.00%
0/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
|
0.00%
0/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/28 • 4 years
|
4.2%
1/24 • 4 years
|
Other adverse events
| Measure |
Everolimus (RAD001)
n=28 participants at risk
Subjects will receive Everolimus 10 mg daily
Everolimus (RAD 001): 10mg of Everolimus or Placebo taken by mouth once daily for 1 year or until progression (whichever comes first).
|
Placebo
n=24 participants at risk
Subjects will receive double-blind placebo
Placebo
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
10.7%
3/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
Alanine aminotransferase increased
|
10.7%
3/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Blood and lymphatic system disorders
Anemia
|
21.4%
6/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Metabolism and nutrition disorders
Anorexia
|
28.6%
8/28 • 4 years
|
12.5%
3/24 • 4 years
|
|
Investigations
Aspartate aminotransferase increased
|
10.7%
3/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Investigations
Cholesterol high
|
32.1%
9/28 • 4 years
|
12.5%
3/24 • 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
4/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
Creatinine increased
|
7.1%
2/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
10.7%
3/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Gastrointestinal disorders
Dry mouth
|
28.6%
8/28 • 4 years
|
25.0%
6/24 • 4 years
|
|
Nervous system disorders
Dysgeusia
|
10.7%
3/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Gastrointestinal disorders
Dysphagia
|
7.1%
2/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.1%
2/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Ear and labyrinth disorders
Ear pain
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
General disorders
Facial pain
|
10.7%
3/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
General disorders
Fatigue
|
25.0%
7/28 • 4 years
|
16.7%
4/24 • 4 years
|
|
General disorders
Fever
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Nervous system disorders
Headache
|
14.3%
4/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
14.3%
4/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
25.0%
7/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
10.7%
3/28 • 4 years
|
25.0%
6/24 • 4 years
|
|
Infections and infestations
Infections and infestations - Other, specify
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Psychiatric disorders
Insomnia
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
Investigations - Other, specify
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
Lymphocyte count decreased
|
32.1%
9/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Gastrointestinal disorders
Nausea
|
14.3%
4/28 • 4 years
|
12.5%
3/24 • 4 years
|
|
Investigations
Neutrophil count decreased
|
14.3%
4/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
General disorders
Pain
|
10.7%
3/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Infections and infestations
Papulopustular rash
|
10.7%
3/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
Platelet count decreased
|
25.0%
7/28 • 4 years
|
4.2%
1/24 • 4 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
10.7%
3/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Infections and infestations
Sinusitis
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
7.1%
2/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Infections and infestations
Skin infection
|
10.7%
3/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Infections and infestations
Pharyngitis
|
46.4%
13/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Ear and labyrinth disorders
Tinnitus
|
7.1%
2/28 • 4 years
|
0.00%
0/24 • 4 years
|
|
Investigations
White blood cell decreased
|
35.7%
10/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
0.00%
0/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
3.6%
1/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
3.6%
1/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
Investigations
Upper respiratory infection
|
0.00%
0/28 • 4 years
|
8.3%
2/24 • 4 years
|
|
General disorders
Vomiting
|
3.6%
1/28 • 4 years
|
8.3%
2/24 • 4 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place