Trial Outcomes & Findings for A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NCT NCT01667562)
NCT ID: NCT01667562
Last Updated: 2019-12-20
Results Overview
Kaplan Meier estimate of the median PFS was defined as the time at which half of the participants have progressed (progressive disease \[PD\]) based on RECIST tumor response criteria or died from any cause, whichever occurred first. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. Patients who had not died or progressed at the time of the final analysis were censored at the date of last contact.
COMPLETED
PHASE3
375 participants
Baseline until disease progression, or death, whichever occurs first (approximately up to 4 years and 9 months)
2019-12-20
Participant Flow
375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
Participant milestones
| Measure |
Diagnostic Phase
Participants with advanced or metastatic NSCLC were tested for EGFR mutations. Participants who did not have an EGFR mutation were excluded from the study.
|
Erlotinib
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|---|
|
Diagnostic Phase
STARTED
|
375
|
0
|
|
Diagnostic Phase
COMPLETED
|
345
|
0
|
|
Diagnostic Phase
NOT COMPLETED
|
30
|
0
|
|
Erlotinib Phase
STARTED
|
0
|
30
|
|
Erlotinib Phase
COMPLETED
|
0
|
27
|
|
Erlotinib Phase
NOT COMPLETED
|
0
|
3
|
Reasons for withdrawal
| Measure |
Diagnostic Phase
Participants with advanced or metastatic NSCLC were tested for EGFR mutations. Participants who did not have an EGFR mutation were excluded from the study.
|
Erlotinib
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|---|
|
Diagnostic Phase
Started Erlotinib in Treatment Period
|
30
|
0
|
|
Erlotinib Phase
Lost to Follow-up
|
0
|
1
|
|
Erlotinib Phase
Adverse Event
|
0
|
1
|
|
Erlotinib Phase
Switch to Commercial Drug
|
0
|
1
|
Baseline Characteristics
375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
Baseline characteristics by cohort
| Measure |
Baseline Population
n=375 Participants
Participants with advanced or metastatic NSCLC were tested for EGFR mutations and enrolled in the study.
|
|---|---|
|
Age, Continuous
Diagnostic Phase
|
62.2 years
STANDARD_DEVIATION 9.2 • n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Age, Continuous
Erlotinib
|
62.4 years
STANDARD_DEVIATION 9.7 • n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Sex: Female, Male
Diagnostic Phase · Female
|
133 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Sex: Female, Male
Diagnostic Phase · Male
|
242 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Sex: Female, Male
Erlotinib · Female
|
18 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Sex: Female, Male
Erlotinib · Male
|
12 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · American Indian or Alaska Native
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · Asian
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · Black or African American
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · White
|
375 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · More than one race
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Diagnostic Phase · Unknown or Not Reported
|
0 Participants
n=375 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · American Indian or Alaska Native
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · Asian
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · Black or African American
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · White
|
30 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · More than one race
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
|
Race (NIH/OMB)
Erlotinib · Unknown or Not Reported
|
0 Participants
n=30 Participants • 375 participants were in the diagnostic population. Out of the 375 participants, only 30 participants had EGFR mutation and were eligible for erlotinib treatment.
|
PRIMARY outcome
Timeframe: Baseline until disease progression, or death, whichever occurs first (approximately up to 4 years and 9 months)Population: The intent-to-treat population included all participants enrolled in the study. There were no patients excluded from analysis either in efficacy and safety analysis.
Kaplan Meier estimate of the median PFS was defined as the time at which half of the participants have progressed (progressive disease \[PD\]) based on RECIST tumor response criteria or died from any cause, whichever occurred first. PD: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. Patients who had not died or progressed at the time of the final analysis were censored at the date of last contact.
Outcome measures
| Measure |
Erlotinib
n=30 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Progression-Free Survival as Assessed by Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 (v 1.1)
|
9.574 months
Interval 5.525 to 13.662
|
SECONDARY outcome
Timeframe: Baseline until disease progression, or death, whichever occurs first (approximately up to 4 years and 9 months)Population: The intent-to-treat population included all participants enrolled in the study. There were no patients excluded from analysis either in efficacy and safety analysis.
Objective response (OR) was based on criteria related to changes in size of target lesions according to modified RECIST. Target lesions were selected on the basis of their size (lesions with the longest diameter) as well as the feasibility of reproducible repeated measurements. OR was the sum of complete response (CR) and partial response (PR) four at least 4 weeks during treatment. CR: disappearance of all target lesions. PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters.
Outcome measures
| Measure |
Erlotinib
n=30 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Proportion of Participants With Objective Response as Assessed by RECIST v 1.1
|
0.67 proportion of participants
Interval 0.49 to 0.81
|
SECONDARY outcome
Timeframe: Baseline until disease progression, or death, whichever occurs first (approximately up to 4 years and 9 months)Population: The intent-to-treat population included all participants enrolled in the study. There were no patients excluded from analysis either in efficacy and safety analysis.
Disease control was defined as objective response or stable disease (SD) for at least 6 weeks. OR was based on criteria related to changes in size of target lesions according to modified RECIST. Target lesions were selected on the basis of their size (lesions with the longest diameter) as well as the feasibility of reproducible repeated measurements. OR was the sum of CR and PR four at least 4 weeks during treatment. CR: disappearance of all target lesions. PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Outcome measures
| Measure |
Erlotinib
n=30 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Proportion of Participants With Disease Control as Assessed by RECIST v 1.1
|
0.97 proportion of participants
Interval 0.82 to 1.0
|
SECONDARY outcome
Timeframe: Screening up to approximately 7 daysPopulation: This study had 2 phases: 1st phase - included 375 patients assessed for EGFR mutations 30 patients (out of these 375) had EGFR mutations and they were included in 2nd phase - treatment with Erlotinib.
Mutations in the EGFR included exon 19 deletion mutations and the single-point substitution mutation L858R in exon 21.
Outcome measures
| Measure |
Erlotinib
n=375 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Proportion of Participants With Epidermal Growth Factor Receptor (EGFR) Mutations
|
0.08 proportion of participants
Interval 0.06 to 0.11
|
SECONDARY outcome
Timeframe: Baseline up to approximately 4 years and 9 monthsPopulation: The safety population was identical to the ITT population, which included all participants enrolled in the study.
An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
Outcome measures
| Measure |
Erlotinib
n=30 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Percentage of Participants With Adverse Events
|
76.7 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline and end of study (approximately 4 years and 9 months)Population: Participants without disease progression who filled out the FACT-L questionnaire.
The domains in the Quality of life score using the Functional Assessment of Cancer Therapy Lung (FACT-L) include physical, social/family, emotional, and functional well-being, and a lung cancer subscale include symptoms, cognitive function and regret of smoking. Minimum and maximum value of the scale is 0 and 4, respectively. Higher score indicate better health state.
Outcome measures
| Measure |
Erlotinib
n=1 Participants
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Change From Baseline to End of Study in Quality of Life Score Using The Functional Assessment of Cancer Therapy Lung (FACT-L)
|
-2.83 unit on a scale
Standard Deviation NA
The standard deviation could not be calculated for one participant who had data for this outcome.
|
Adverse Events
Erlotinib
Serious adverse events
| Measure |
Erlotinib
n=30 participants at risk
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
3/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Skin and subcutaneous tissue disorders
Paronychia
|
3.3%
1/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
General disorders
Death
|
3.3%
1/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
3.3%
1/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
Other adverse events
| Measure |
Erlotinib
n=30 participants at risk
Erlotinib was administered as a single daily oral dose of 150 milligrams until disease progression, death or unacceptable toxicity.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
46.7%
14/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
6.7%
2/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
General disorders
Fatigue
|
13.3%
4/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Blood and lymphatic system disorders
Anemia
|
10.0%
3/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
10.0%
3/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
3/30 • 4 years and 9 months
The safety population was identical to the ITT population. The ITT population comprised all 30 patients.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER