Trial Outcomes & Findings for A Study of Erlotinib (Tarceva) in Participants With Non-Small Cell Lung Cancer (NSCLC) (NCT NCT02774278)
NCT ID: NCT02774278
Last Updated: 2016-08-08
Results Overview
Affymetrix gene expression profiles were primarily analyzed to identify differentially expressed genes between the participants who derived clinical benefit status and those who did not. Analysis was performed using a multivariate linear model (with clinical benefit status, histology, ethnicity, sex, RNA integrity number (RIN), smoking status and stage as predictors), and a False Discovery Rate criteria of below 0.3 as cut-off. Clinical benefit is defined in the Outcome Measure 5.
COMPLETED
PHASE2
264 participants
Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 years
2016-08-08
Participant Flow
Participant milestones
| Measure |
Erlotinib
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Overall Study
STARTED
|
264
|
|
Overall Study
Safety Analysis Population (SAP)
|
261
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
264
|
Reasons for withdrawal
| Measure |
Erlotinib
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Overall Study
Lack of Efficacy
|
199
|
|
Overall Study
Death
|
29
|
|
Overall Study
Adverse event/intercurrent illness
|
16
|
|
Overall Study
Refused treatment/did not cooperate
|
9
|
|
Overall Study
Administrative reasons
|
6
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Protocol Violation
|
2
|
Baseline Characteristics
A Study of Erlotinib (Tarceva) in Participants With Non-Small Cell Lung Cancer (NSCLC)
Baseline characteristics by cohort
| Measure |
Erlotinib
n=264 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Age, Continuous
|
60.8 years
STANDARD_DEVIATION 10.47 • n=5 Participants
|
|
Sex: Female, Male
Female
|
80 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
184 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 yearsPopulation: Primary analysis set (PAS) included all participants who provided evaluable tissue samples and were included in the primary Affymetrix efficacy analysis.
Affymetrix gene expression profiles were primarily analyzed to identify differentially expressed genes between the participants who derived clinical benefit status and those who did not. Analysis was performed using a multivariate linear model (with clinical benefit status, histology, ethnicity, sex, RNA integrity number (RIN), smoking status and stage as predictors), and a False Discovery Rate criteria of below 0.3 as cut-off. Clinical benefit is defined in the Outcome Measure 5.
Outcome measures
| Measure |
Erlotinib
n=102 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Number of Differentially Expressed Genes Associated With Clinical Benefit
|
0 genes
|
PRIMARY outcome
Timeframe: Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 yearsPopulation: PAS participants who had EGFR mutation at baseline were included in this analysis.
Affymetrix gene expression profiles were primarily analyzed to identify differentially expressed genes between the participants who derived clinical benefit status and those who did not. Analysis was performed using a multivariate linear model (with clinical benefit status, histology, ethnicity, sex, RNA integrity number (RIN), smoking status and stage as predictors), and a False Discovery Rate criteria of below 0.3 as cut-off. Number of participants with EGFR mutation (L858R/ exon 19 deletion) and who achieved clinical benefit status was reported. Clinical benefit is defined in the Outcome Measure 5.
Outcome measures
| Measure |
Erlotinib
n=6 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Number of Epidermal Growth Factor Receptor (EGFR) Mutation Participants Who Achieved Clinical Benefit
|
6 participants
|
PRIMARY outcome
Timeframe: Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 yearsPopulation: PAS participants who had KRAS mutation at baseline were included in this analysis.
Affymetrix gene expression profiles were primarily analyzed to identify differentially expressed genes between the participants who derived clinical benefit status and those who did not. Analysis was performed using a multivariate linear model (with clinical benefit status, histology, ethnicity, sex, RNA integrity number (RIN), smoking status and stage as predictors), and a False Discovery Rate criteria of below 0.3 as cut-off. Number of participants with KRAS gene mutation and who achieved clinical benefit status was reported. Clinical benefit is defined in the Outcome Measure 5.
Outcome measures
| Measure |
Erlotinib
n=10 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Number of KRAS Mutation Participants Who Achieved Clinical Benefit
|
4 participants
|
SECONDARY outcome
Timeframe: Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 yearsPopulation: Analysis population included all enrolled participants.
Tumor response was defined as either a CR or a PR prior to failure (disease progression, death from any cause, or a second malignancy). CR was defined as the complete disappearance on magnetic response imaging of all enhancing tumor and mass effect on a stable or decreasing dose of dexamethasone (or only receiving adrenal replacement doses) accompanied by a stable or improving neurologic examination that was maintained for at least 12 weeks. PR was defined as a greater than or equal to 50 percent (%) reduction in tumor size by bi-dimensional measurement on a stable or decreasing dose of dexamethasone accompanied by a stable or improving neurologic examination that was maintained for at least 12 weeks.
Outcome measures
| Measure |
Erlotinib
n=264 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Percentage of Participants With Overall Response of Complete Response (CR) or Partial Response (PR) Using Response Evaluation Criteria in Solid Tumors (RECIST)
|
15.2 percentage of participants
Interval 11.1 to 20.1
|
SECONDARY outcome
Timeframe: Baseline until disease progression, unacceptable toxicity or death, evaluated up to 4 yearsPopulation: Analysis population included all enrolled participants.
Clinical benefit was defined as either a CR, PR, or SD prior to failure (disease progression, death from any cause, or a second malignancy). CR: complete disappearance on magnetic response imaging of all enhancing tumor and mass effect on a stable or decreasing dose of dexamethasone (or only receiving adrenal replacement doses) accompanied by a stable or improving neurologic examination that was maintained for at least 12 weeks. PR: greater than or equal to 50% reduction in tumor size by bi-dimensional measurement on a stable or decreasing dose of dexamethasone accompanied by a stable or improving neurologic examination that was maintained for at least 12 weeks. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD) taking as reference smallest sum of longest dimensions since treatment started associated to non-progressive disease response for non-target lesions. PD: unequivocal progression of existing non-target lesions.
Outcome measures
| Measure |
Erlotinib
n=264 Participants
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Percentage of Participants With Clinical Benefit (CR, PR, or Stable Disease [SD] for at Least 12 Weeks After Study Entry) Using RECIST
|
31.4 percentage of participants
Interval 25.9 to 37.4
|
Adverse Events
Erlotinib
Serious adverse events
| Measure |
Erlotinib
n=261 participants at risk
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
4.2%
11/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
1.9%
5/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.77%
2/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Hydropneumothorax
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Pneumonia
|
4.6%
12/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Urinary tract infection
|
0.77%
2/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Bronchitis
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Pneumonia staphylococcal
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Infections and infestations
Septic shock
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Asthenia
|
1.1%
3/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
General physical health deterioration
|
1.1%
3/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Pyrexia
|
1.1%
3/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Fatigue
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Gait disturbance
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Diarrhoea
|
1.1%
3/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Vomiting
|
0.77%
2/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Dysphagia
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Gastric ulcer
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Cardiac disorders
Acute myocardial infarction
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Cardiac disorders
Cardiac failure congestive
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Cardiac disorders
Myocardial ischaemia
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Cardiac disorders
Pericarditis
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Nervous system disorders
Cerebral infarction
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Nervous system disorders
Cerebrovascular accident
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Nervous system disorders
Dizziness
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Nervous system disorders
Ischaemic stroke
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.77%
2/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Blood and lymphatic system disorders
Haemorrhagic diathesis
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.77%
2/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin ulcer haemorrhage
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Psychiatric disorders
Agitation
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Psychiatric disorders
Completed suicide
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Psychiatric disorders
Confusional state
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Vascular disorders
Arterial haemorrhage
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Vascular disorders
Shock
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Injury, poisoning and procedural complications
Fall
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Renal and urinary disorders
Renal failure acute
|
0.38%
1/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
Other adverse events
| Measure |
Erlotinib
n=261 participants at risk
Erlotinib was administered at 150 milligrams (mg) orally daily until disease progression, unacceptable toxicity or death.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
51.0%
133/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.6%
33/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.3%
32/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.4%
14/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Diarrhoea
|
32.2%
84/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
26/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Gastrointestinal disorders
Nausea
|
8.8%
23/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.9%
39/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
14.2%
37/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
5.7%
15/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Fatigue
|
12.3%
32/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
General disorders
Pyrexia
|
5.4%
14/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
|
Metabolism and nutrition disorders
Anorexia
|
15.7%
41/261 • Up to 4 years
Safety analysis population (SAP) included all participants who received at least one dose of the trial medication and had a safety follow-up.
|
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