Trial Outcomes & Findings for A Study of Tarceva (Erlotinib) in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer Who Present EGFR Mutations (NCT NCT01287754)

NCT ID: NCT01287754

Last Updated: 2015-06-02

Results Overview

PFS was defined as the time from the first dose of erlotinib to the first documentation of disease progression or death, whichever occurred first. Tumor progression was determined using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), which defines progression as a 20 percent (%) or greater increase in the sum of diameters of target lesions with an absolute increase of at least 5 millimeters (mm), or the appearance of one or more new lesions. PFS was calculated in months as \[first event date minus first dose date plus 1\] divided by 30.44.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 24 participants
• Primary outcome timeframe: Per standard of care (every 3 months) until discontinuation for up to approximately 2 years
• Results posted on: 2015-06-02

Participant Flow

All participants underwent EGFR mutation testing at Screening. Those positive for the EGFR mutation and who met eligibility criteria (number of participants [n] = 3) received treatment with erlotinib. The remaining participants (n = 21) were followed for overall survival but did not receive treatment.

Participant milestones

Measure	Erlotinib Participants positive for the epidermal growth factor receptor (EGFR) mutation and who met eligibility criteria received treatment with erlotinib, 150 milligrams (mg) orally once daily until disease progression or unacceptable toxicity.	Untreated Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Overall Study STARTED	3	21
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	3	21

Reasons for withdrawal

Measure	Erlotinib Participants positive for the epidermal growth factor receptor (EGFR) mutation and who met eligibility criteria received treatment with erlotinib, 150 milligrams (mg) orally once daily until disease progression or unacceptable toxicity.	Untreated Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Overall Study Death	2	12
Overall Study Lost to Follow-up	1	6
Overall Study Protocol Violation	0	3

Baseline Characteristics

A Study of Tarceva (Erlotinib) in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer Who Present EGFR Mutations

Baseline characteristics by cohort

Measure	Erlotinib n=3 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated n=21 Participants Participants without the EGFR mutation were followed for overall survival but did not undergo treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.	Total n=24 Participants Total of all reporting groups
Age, Continuous	75 years STANDARD_DEVIATION 7.6 • n=93 Participants	67 years STANDARD_DEVIATION 8.4 • n=4 Participants	68 years STANDARD_DEVIATION 8.5 • n=27 Participants
Sex: Female, Male Female	2 Participants n=93 Participants	13 Participants n=4 Participants	15 Participants n=27 Participants
Sex: Female, Male Male	1 Participants n=93 Participants	8 Participants n=4 Participants	9 Participants n=27 Participants

PRIMARY outcome

Timeframe: Per standard of care (every 3 months) until discontinuation for up to approximately 2 years

Population: All Participants Treated: All participants who received at least one dose of erlotinib were included in the analysis.

PFS was defined as the time from the first dose of erlotinib to the first documentation of disease progression or death, whichever occurred first. Tumor progression was determined using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), which defines progression as a 20 percent (%) or greater increase in the sum of diameters of target lesions with an absolute increase of at least 5 millimeters (mm), or the appearance of one or more new lesions. PFS was calculated in months as [first event date minus first dose date plus 1] divided by 30.44.

Outcome measures

Measure	Erlotinib n=3 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Progression-Free Survival (PFS) Among Erlotinib-Treated Participants With the EGFR Mutation	13.7 months Interval 2.0 to Data for 1 of 3 participants were censored, and thus a confidence interval upper limit was not reached.	—

SECONDARY outcome

Timeframe: Per standard of care (every 3 months) until discontinuation for up to approximately 2 years

Population: All Participants Treated

Objective tumor response was assessed by the investigator using RECIST v1.1 and recorded as complete response (CR), partial response (PR), or unmeasurable. RECIST v1.1 defines CR as disappearance of all target lesions, with short-axis reduction to less than (<) 10 mm for any pathological lymph nodes, and PR as a 30% or greater reduction from baseline in the sum of diameters of target lesions.

Outcome measures

Measure	Erlotinib n=3 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Number of Erlotinib-Treated Participants With the EGFR Mutation With an Objective Response Per RECIST v1.1 Partial response	2 participants	—
Number of Erlotinib-Treated Participants With the EGFR Mutation With an Objective Response Per RECIST v1.1 Unmeasurable	1 participants	—

SECONDARY outcome

Timeframe: Per standard of care (every 3 months) until discontinuation for up to approximately 2 years

Population: All Participants Enrolled: All erlotinib-treated participants who received at least one dose of erlotinib, in addition to all enrolled untreated participants, were included in the analysis.

OS was defined as the time from recorded diagnosis to death from any cause or last patient last visit. OS was calculated in months as [death date or last-known alive date minus diagnosis date plus 1] divided by 30.44.

Outcome measures

Measure	Erlotinib n=3 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated n=21 Participants Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Overall Survival (OS) Among Erlotinib-Treated and Untreated Participants	17.8 months Interval 17.6 to Data for 1 of 3 participants were censored, and thus a confidence interval upper limit was not reached.	11.3 months Interval 8.4 to 11.4

SECONDARY outcome

Timeframe: At 6 and 12 months

Population: All Participants Enrolled

Death from any cause was documented at 6 and 12 months from recorded diagnosis. The percentage of participants alive at each timepoint was calculated as [number of participants alive divided by number enrolled] multiplied by 100.

Outcome measures

Measure	Erlotinib n=3 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated n=21 Participants Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Percentage of Participants Alive at 6 and 12 Months At 6 months	100 percentage of participants	67 percentage of participants
Percentage of Participants Alive at 6 and 12 Months At 12 months	100 percentage of participants	24 percentage of participants

SECONDARY outcome

Timeframe: Screening

Population: All Participants Enrolled

Participants were tested at Screening for the presence of activating mutations in the tyrosine kinase domain of EGFR. The percentage of participants with mutation was calculated as [number of mutation-positive participants divided by number tested] multiplied by 100.

Outcome measures

Measure	Erlotinib n=24 Participants Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.	Untreated Participants without the EGFR mutation were followed for overall survival but did not receive treatment. Additionally, participants positive for the EGFR mutation who were excluded from treatment were followed for overall survival.
Percentage of Participants With EGFR Mutation at Screening	17 percentage of participants Interval 2.0 to 32.0	—

Adverse Events

Erlotinib

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	Erlotinib n=3 participants at risk Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.
Infections and infestations Epidural catheter infection	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.

Other adverse events

Measure	Erlotinib n=3 participants at risk Participants positive for the EGFR mutation and who met eligibility criteria received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.
Skin and subcutaneous tissue disorders Alopecia	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.
Gastrointestinal disorders Bleeding from mouth	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.
Infections and infestations Eye infection	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lipoma	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Periosteal reaction	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.
Nervous system disorders Paraparesis	33.3% 1/3 • Up to 2 years Only participants treated with erlotinib were assessed for adverse events.

Additional Information

Medical Communications

Hoffmann-LaRoche

Phone: 800-821-8590

Email: genentech@druginfo.com

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