Trial Outcomes & Findings for A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor Mutations (NCT NCT01260181)

Last Updated: 2018-10-31

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

Baseline up to 5 years (assessed at Baseline, every 8 weeks until disease progression or death or end of treatment period [up to 5 years])

Results posted on

2018-10-31

Participant Flow

Participant milestones

Participant milestones
Measure	Erlotinib Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Erlotinib Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Overall Study Decision of Investigator	1

Baseline Characteristics

A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor Mutations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Age, Continuous	66.33 years STANDARD_DEVIATION 9.21 • n=93 Participants
Sex: Female, Male Female	24 Participants n=93 Participants
Sex: Female, Male Male	6 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	0 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	30 Participants n=93 Participants

PRIMARY outcome

Timeframe: Baseline up to 5 years (assessed at Baseline, every 8 weeks until disease progression or death or end of treatment period [up to 5 years])

Population: The ITT population was defined as all subjects who are enrolled to the treatment phase of the study, regardless if they completed treatment.

Outcome measures

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Percentage of Participants With Objective Response (Complete Response [CR]/Partial Response [PR]) Based on Computer Tomography (CT) or Magnetic Resonance Imaging (MRI) According to Response Evaluation Criteria in Solid Tumors (RECIST) Version (v) 1.1	63.3 percentage of participants Interval 46.1 to 80.6

SECONDARY outcome

Timeframe: Baseline up to 5 years (assessed at Baseline, every 8 weeks until disease progression or death or end of treatment period [up to 5 years])

Population: The ITT population was defined as all subjects who are enrolled to the treatment phase of the study, regardless if they completed treatment.

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

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Progression Free Survival (PFS) Based on CT or MRI According to RECIST v 1.1	40 weeks Interval 31.0 to 63.0

SECONDARY outcome

Timeframe: Baseline up to 5 years

Population: The ITT population was defined as all subjects who are enrolled to the treatment phase of the study, regardless if they completed treatment.

Overall Survival (OS) was defined as the time between the date of randomization and the date of death due to any cause.

Outcome measures

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Overall Survival	83 weeks Interval 56.0 to 125.0

SECONDARY outcome

Timeframe: Baseline up to 5 years

Population: 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

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Percentage of Participants With Adverse Events	29 percentage of participants

SECONDARY outcome

Timeframe: Screening (21 days prior to Day 1)

Population: The ITT population was defined as all subjects who are enrolled to the treatment phase of the study, regardless if they completed treatment.

Mutations in the EGFR included exon 19 deletion mutations and the single-point substitution mutation L858R in exon 21.

Outcome measures

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Percentage of Participants With Epidermal Growth Factor Receptor (EGFR) Mutation in Study Population Exon 19 mutation	40 percentage of participants
Percentage of Participants With Epidermal Growth Factor Receptor (EGFR) Mutation in Study Population Exon 21 mutation	60 percentage of participants

SECONDARY outcome

Timeframe: Baseline up to 5 years (assessed at Baseline, every 8 weeks until disease progression or death or end of treatment period [up to 5 years])

Population: The ITT population was defined as all subjects who are enrolled to the treatment phase of the study, regardless if they completed treatment.

The response duration was defined as the time of initial response (complete response (CR) /partial response (PR) whichever is first recorded) until documented disease progression. CR was defined as disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial response was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression was defined as 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).

Outcome measures

Outcome measures
Measure	Erlotinib n=30 Participants Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Median Time Taken From the First Response Until Disease Progression Based on RECIST v 1.1 as Determined by the Investigator	41.5 weeks Interval 32.0 to 63.0

Adverse Events

Erlotinib

Serious events: 8 serious events

Other events: 29 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	Erlotinib n=30 participants at risk Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
General disorders Death	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Diarrhoea	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Pneumonia	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Intestinal Perforation	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Injury, poisoning and procedural complications Fracture	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Pelvic Infection	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Rhabdomyolysis	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Pulmonary Embolism	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Rash	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Psychiatric disorders Depression	3.3% 1/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.

Other adverse events

Other adverse events
Measure	Erlotinib n=30 participants at risk Participants received erlotinib 150 millgrams (mg) orally daily until disease progression.
Skin and subcutaneous tissue disorders Dermatitis Acneiform	63.3% 19/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Rash Maculo-Papular	26.7% 8/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Alopecia	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Acne	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Erythema	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Diarrhoea	50.0% 15/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Constipation	20.0% 6/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Nausea	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Stomatitis	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Dyspepsia	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Abdominal Pain	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Odynophagia	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Gastrointestinal disorders Vomiting	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Paronychia	23.3% 7/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Upper Respiratory Tract Infection	23.3% 7/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Eye Infection	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Infections and infestations Conjunctivitis	20.0% 6/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
General disorders Fatigue	23.3% 7/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
General disorders Oedema Peripheral	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
General disorders Influenza Like Illness	13.3% 4/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
General disorders Asthenia	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
General disorders Pain	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Back Pain	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Pain in Extremity	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Bone Pain	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Musculoskeletal Chest Pain	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Musculoskeletal and connective tissue disorders Neck Pain	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Cough	13.3% 4/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Dyspnoea	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Haemoptysis	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Respiratory, thoracic and mediastinal disorders Productive Cough	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Eye disorders Dry Eye	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Eye disorders Blepharitis	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Eye disorders Cataract	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Psychiatric disorders Anxiety	13.3% 4/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Psychiatric disorders Depression	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Psychiatric disorders Insomnia	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Metabolism and nutrition disorders Decreased Appetite	26.7% 8/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Metabolism and nutrition disorders Hyperglycaemia	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Metabolism and nutrition disorders Hypomagnesaemia	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Nervous system disorders Headache	13.3% 4/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Nervous system disorders Dizziness	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Investigations Blood Bilirubin Increased	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Investigations Neutrophil Count Increased	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Investigations Weight Decreased	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Renal and urinary disorders Pollakiuria	10.0% 3/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Blood and lymphatic system disorders Leukocytosis	6.7% 2/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.
Skin and subcutaneous tissue disorders Pruritus	16.7% 5/30 • 5 years The safety population was identical to the ITT population, which included all participants enrolled in the study.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: [email protected]

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