Non-Small Cell Lung Cancer
Conditions
Brief summary
This study will provide treatment with erlotinib to participants with advanced NSCLC who have received at least one course of standard chemotherapy or radiation therapy, or who are not medically suitable for either. Efficacy and safety will be monitored throughout the study.
Interventions
DRUGErlotinib
Erlotinib will be given orally as 150 milligrams (mg) once daily until unacceptable toxicity, disease progression, or withdrawal for any other reason.
Sponsors
Hoffmann-La Roche
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adults greater than or equal to (≥) 18 years of age * Histologically or cytologically documented inoperable, locally advanced, metastatic, or recurrent NSCLC * Previous treatment with no more than 2 prior chemotherapy regimens
Exclusion criteria
* Previous systemic anti-cancer therapy with human epidermal growth factor receptor 1 (HER1)/epidermal growth factor receptor (EGFR) inhibitors * Inability to take oral medication * Any other malignancies within 5 years
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Objective Response According to Response Evaluation Criteria in Solid Tumors (RECIST) | Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter | Objective response was defined as a best overall response of either complete response (CR) or partial response (PR) as assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as greater than or equal to (≥) 30 percent (%) decrease in sum of longest diameter (LD) of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. The percentage of participants (in nearest integer) with objective response was reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants by Best Overall Response According to RECIST | Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter | Tumor response was assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as ≥30% decrease in sum LD of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. SD was defined as neither sufficient shrinkage to qualify for PR but \<20% increase in sum LD. Disease progression or progressive disease (PD) was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. The percentage of participants (in nearest integer unless the percentage is \<1) with each type of best overall response was reported. |
| Percentage of Participants With Death or Disease Progression According to RECIST | Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter | Tumor response was assessed by RECIST during the study. Disease progression or PD was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. The percentage of participants (in nearest integer) who died or experienced PD was reported. |
| Percentage of Participants With Disease Control According to RECIST | Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter | Disease control was defined as a best overall response of either CR, PR, or stable disease (SD) as assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as ≥30% decrease in sum LD of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. SD was defined as neither sufficient shrinkage to qualify for PR but less than (\<) 20% increase in sum LD. The percentage of participants (in nearest integer) with disease control was reported. |
| Percentage of Participants Who Died | Up to approximately 4.5 years; assessed continuously during treatment (up to 3.5 years) and every 6 months thereafter | The percentage of participants (in nearest integer) who died from any cause was reported. |
| Overall Survival (OS) | Up to approximately 4.5 years; assessed continuously during treatment (up to 3.5 years) and every 6 months thereafter | OS was defined as the time from start of treatment to date of death for any reason. The median duration of OS and corresponding 95% CI were estimated by Kaplan-Meier analysis and expressed in months. |
| Progression-Free Survival (PFS) According to RECIST | Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter | Tumor response was assessed by RECIST during the study. Disease progression or PD was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. PFS was defined as the time from start of treatment to the first event of death or PD. The median duration of PFS and corresponding 95% confidence interval (CI) were estimated by Kaplan-Meier analysis and expressed in months. |
Countries
Albania, Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Brazil, Bulgaria, Chile, China, Colombia, Croatia, Czechia, Ecuador, Egypt, Estonia, Finland, Germany, Greece, Guatemala, Hong Kong, Hungary, India, Indonesia, Ireland, Israel, Italy, Latvia, Lithuania, Malaysia, Mexico, Netherlands, New Zealand, Panama, Peru, Poland, Portugal, Romania, Russia, Saudi Arabia, Serbia, Slovakia, Slovenia, South Korea, Sweden, Switzerland, Taiwan, Thailand, Turkey (Türkiye), Uruguay, Venezuela
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Erlotinib Erlotinib was given as a single agent in this EAP to participants with inoperable, locally advanced, recurrent, or metastatic NSCLC. Participants were treated with 150 mg oral erlotinib once daily until unacceptable toxicity, disease progression, or withdrawal for any other reason. The dose could be reduced in the event of toxicity, and some participants therefore received 50 or 100 mg once daily. The study completed after every participant had either died or been followed for 6 months after stopping erlotinib. | 6,586 |
| Total | 6,586 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 83 |
| Overall Study | Death Due to Adverse Event | 115 |
| Overall Study | Death Due to Malignant Disease | 456 |
| Overall Study | Death Due to Progression/Deterioration | 4 |
| Overall Study | Death Due to Toxicity | 3 |
| Overall Study | Death from Unknown Cause | 5 |
| Overall Study | Logistical Reasons | 13 |
| Overall Study | Lost to Follow-up | 100 |
| Overall Study | No Data | 9 |
| Overall Study | Non-Compliance | 10 |
| Overall Study | Other | 4 |
| Overall Study | Participant Refusal | 390 |
| Overall Study | Physician/Participant Decision | 7 |
| Overall Study | Progressive Disease | 3,750 |
| Overall Study | Prolonged Dosing Interruption | 4 |
| Overall Study | Serious Adverse Event | 6 |
| Overall Study | Study Drug-Related Adverse Event | 315 |
| Overall Study | Switched to Commercial Treatment | 4 |
| Overall Study | Switched to Compassionate/Other Use | 174 |
| Overall Study | Symptomatic Deterioration | 1,088 |
| Overall Study | Unspecified Progression/Deterioration | 13 |
| Overall Study | Violation of Eligibility | 23 |
Baseline characteristics
| Characteristic | Erlotinib |
|---|---|
| Age, Continuous | 62.4 years STANDARD_DEVIATION 11.24 |
| Sex: Female, Male Female | 2608 Participants |
| Sex: Female, Male Male | 3978 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 6,586 |
| serious Total, serious adverse events | 2,983 / 6,586 |
Outcome results
Primary
Percentage of Participants With Objective Response According to Response Evaluation Criteria in Solid Tumors (RECIST)
Objective response was defined as a best overall response of either complete response (CR) or partial response (PR) as assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as greater than or equal to (≥) 30 percent (%) decrease in sum of longest diameter (LD) of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. The percentage of participants (in nearest integer) with objective response was reported.
Time frame: Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter
Population: ITT/Safety Population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Erlotinib | Percentage of Participants With Objective Response According to Response Evaluation Criteria in Solid Tumors (RECIST) | 11 percentage of participants |
Secondary
Overall Survival (OS)
OS was defined as the time from start of treatment to date of death for any reason. The median duration of OS and corresponding 95% CI were estimated by Kaplan-Meier analysis and expressed in months.
Time frame: Up to approximately 4.5 years; assessed continuously during treatment (up to 3.5 years) and every 6 months thereafter
Population: ITT/Safety Population.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Erlotinib | Overall Survival (OS) | 7.9 months |
Secondary
Percentage of Participants by Best Overall Response According to RECIST
Tumor response was assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as ≥30% decrease in sum LD of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. SD was defined as neither sufficient shrinkage to qualify for PR but \<20% increase in sum LD. Disease progression or progressive disease (PD) was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. The percentage of participants (in nearest integer unless the percentage is \<1) with each type of best overall response was reported.
Time frame: Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter
Population: ITT/Safety Population.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Complete Response (CR) | 0.7 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Partial Response (PR) | 10 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Stable Disease (SD) | 45 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Progressive Disease (PD) | 23 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Not Evaluable | 3 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Not Done | 18 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | Not Known | 0.0 percentage of participants |
| Erlotinib | Percentage of Participants by Best Overall Response According to RECIST | No Data | 0.3 percentage of participants |
Secondary
Percentage of Participants Who Died
The percentage of participants (in nearest integer) who died from any cause was reported.
Time frame: Up to approximately 4.5 years; assessed continuously during treatment (up to 3.5 years) and every 6 months thereafter
Population: ITT/Safety Population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Erlotinib | Percentage of Participants Who Died | 81 percentage of participants |
Secondary
Percentage of Participants With Death or Disease Progression According to RECIST
Tumor response was assessed by RECIST during the study. Disease progression or PD was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. The percentage of participants (in nearest integer) who died or experienced PD was reported.
Time frame: Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter
Population: ITT/Safety Population; only participants with available data were included.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Erlotinib | Percentage of Participants With Death or Disease Progression According to RECIST | 93 percentage of participants |
Secondary
Percentage of Participants With Disease Control According to RECIST
Disease control was defined as a best overall response of either CR, PR, or stable disease (SD) as assessed by RECIST during the study. CR was defined as disappearance of all clinical and radiographic evidence of target and non-target lesions, normal tumor markers, and absence of tumor-related symptoms. PR was defined as ≥30% decrease in sum LD of target lesions in reference to Baseline sum LD. Response was to be confirmed ≥28 days after the initial assessment of CR or PR. SD was defined as neither sufficient shrinkage to qualify for PR but less than (\<) 20% increase in sum LD. The percentage of participants (in nearest integer) with disease control was reported.
Time frame: Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter
Population: ITT/Safety Population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Erlotinib | Percentage of Participants With Disease Control According to RECIST | 56 percentage of participants |
Secondary
Progression-Free Survival (PFS) According to RECIST
Tumor response was assessed by RECIST during the study. Disease progression or PD was defined as ≥20% increase in sum LD in reference to the smallest on-treatment sum LD, or the appearance of new lesions. PFS was defined as the time from start of treatment to the first event of death or PD. The median duration of PFS and corresponding 95% confidence interval (CI) were estimated by Kaplan-Meier analysis and expressed in months.
Time frame: Up to approximately 4.5 years; assessed at Baseline, according to institutional standards during treatment (up to 3.5 years), and every 6 months thereafter
Population: ITT/Safety Population; only participants with available data were included.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Erlotinib | Progression-Free Survival (PFS) According to RECIST | 3.3 months |