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    Clinical Trial Results:
    A Study of Erlotinib (Tarceva®) Treatment in Patients With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Who Present Activating Mutations in the Tyrosine Kinase Domain of the Epidermal Growth Factor Receptor

    Summary
    EudraCT number
    2010-023869-21
    Trial protocol
    FI  
    Global end of trial date
    20 Nov 2013

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Jul 2016
    First version publication date
    06 Aug 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ML25575
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01287754
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    F. Hoffmann-La Roche AG
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, CH-4070
    Public contact
    Roche Trial Information Hotline, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com
    Scientific contact
    Roche Trial Information Hotline, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    03 Nov 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Nov 2013
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Nov 2013
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The study was designed to assess the efficacy of erlotinib (Tarceva[TM]; 150 milligrams [mg]) on progression-free survival (PFS) in patients with non-small cell lung cancer (NSCLC) in locally advanced or metastatic stages (stage IIIB and stage IV) who have not received previous chemotherapy for their disease and who present activating mutations in the tyrosine kinase (TK) domain of the epidermal growth factor receptor (EGFR).
    Protection of trial subjects
    This study was done in accordance with the Declaration of Helsinki and Good Clinical Practice guidelines.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    22 Mar 2011
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Finland: 24
    Worldwide total number of subjects
    24
    EEA total number of subjects
    24
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    11
    From 65 to 84 years
    12
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    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.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Erlotinib
    Arm description
    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.
    Arm type
    Experimental

    Investigational medicinal product name
    Erlotinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.

    Arm title
    Untreated
    Arm description
    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.
    Arm type
    No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    Total Population
    Arm description
    All participants underwent EGFR mutation testing at Screening. Those positive for the EGFR mutation and who met eligibility criteria (n = 3) received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity. The remaining participants (n = 21) were followed for overall survival but did not receive treatment.
    Arm type
    Experimental or no intervention

    Investigational medicinal product name
    Erlotinib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Eligible participants (n = 3) received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity.

    Number of subjects in period 1
    Erlotinib Untreated Total Population
    Started
    3
    21
    24
    Completed
    0
    0
    0
    Not completed
    3
    21
    24
         Protocol deviation
    -
    3
    3
         Death
    2
    12
    14
         Lost to follow-up
    1
    6
    7

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Erlotinib
    Reporting group description
    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.

    Reporting group title
    Untreated
    Reporting group description
    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.

    Reporting group title
    Total Population
    Reporting group description
    All participants underwent EGFR mutation testing at Screening. Those positive for the EGFR mutation and who met eligibility criteria (n = 3) received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity. The remaining participants (n = 21) were followed for overall survival but did not receive treatment.

    Reporting group values
    Erlotinib Untreated Total Population Total
    Number of subjects
    3 21 24
    Age categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    75 ± 7.6 67 ± 8.4 68 ± 8.5 -
    Gender categorical
    Units: Subjects
        Female
    2 13 15 15
        Male
    1 8 9 9

    End points

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    End points reporting groups
    Reporting group title
    Erlotinib
    Reporting group description
    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.

    Reporting group title
    Untreated
    Reporting group description
    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.

    Reporting group title
    Total Population
    Reporting group description
    All participants underwent EGFR mutation testing at Screening. Those positive for the EGFR mutation and who met eligibility criteria (n = 3) received treatment with erlotinib, 150 mg orally once daily until disease progression or unacceptable toxicity. The remaining participants (n = 21) were followed for overall survival but did not receive treatment.

    Primary: Progression-Free Survival (PFS) among Erlotinib-Treated Participants with the EGFR Mutation

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    End point title
    Progression-Free Survival (PFS) among Erlotinib-Treated Participants with the EGFR Mutation [1] [2]
    End point description
    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. All participants who received at least one dose of erlotinib were included in the analysis. (99999 = not estimable because data for 1 of 3 participants were censored, and thus a confidence interval upper limit was not reached.)
    End point type
    Primary
    End point timeframe
    Per standard of care (every 3 months) until discontinuation for up to approximately 2 years
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Statistical analysis was not performed and/or reported because there were no statistical assumptions. Only descriptive statistics were planned.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Erlotinib-treated participants were analyzed for all reported outcome measures; untreated participants were only followed for overall survival and are thus only included in the survival outcome measures. Except for the percentage of participants with EGFR mutation at Screening, outcome measures were not analyzed for the total collective population.
    End point values
    Erlotinib
    Number of subjects analysed
    3
    Units: months
        median (confidence interval 95%)
    13.7 (2 to 99999)
    No statistical analyses for this end point

    Secondary: Number of Erlotinib-Treated Participants with the EGFR Mutation with an Objective Response per RECIST v1.1

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    End point title
    Number of Erlotinib-Treated Participants with the EGFR Mutation with an Objective Response per RECIST v1.1 [3]
    End point description
    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. All participants who received at least one dose of erlotinib were included in the analysis.
    End point type
    Secondary
    End point timeframe
    Per standard of care (every 3 months) until discontinuation for up to approximately 2 years
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Erlotinib-treated participants were analyzed for all reported outcome measures; untreated participants were only followed for overall survival and are thus only included in the survival outcome measures. Except for the percentage of participants with EGFR mutation at Screening, outcome measures were not analyzed for the total collective population.
    End point values
    Erlotinib
    Number of subjects analysed
    3
    Units: participants
        Partial response
    2
        Unmeasurable
    1
    No statistical analyses for this end point

    Secondary: Overall Survival (OS) among Erlotinib-Treated and Untreated Participants

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    End point title
    Overall Survival (OS) among Erlotinib-Treated and Untreated Participants [4]
    End point description
    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. All erlotinib-treated participants who received at least one dose of erlotinib, in addition to all enrolled untreated participants, were included in the analysis. (99999 = not estimable because data for 1 of 3 participants were censored, and thus a confidence interval upper limit was not reached.)
    End point type
    Secondary
    End point timeframe
    Per standard of care (every 3 months) until discontinuation for up to approximately 2 years
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Erlotinib-treated participants were analyzed for all reported outcome measures; untreated participants were only followed for overall survival and are thus only included in the survival outcome measures. Except for the percentage of participants with EGFR mutation at Screening, outcome measures were not analyzed for the total collective population.
    End point values
    Erlotinib Untreated
    Number of subjects analysed
    3
    21
    Units: months
        median (confidence interval 95%)
    17.8 (17.6 to 99999)
    11.3 (8.4 to 11.4)
    No statistical analyses for this end point

    Secondary: Percentage of Participants Alive at 6 and 12 Months

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    End point title
    Percentage of Participants Alive at 6 and 12 Months [5]
    End point description
    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. All erlotinib-treated participants who received at least one dose of erlotinib, in addition to all enrolled untreated participants, were included in the analysis.
    End point type
    Secondary
    End point timeframe
    At 6 and 12 months
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Erlotinib-treated participants were analyzed for all reported outcome measures; untreated participants were only followed for overall survival and are thus only included in the survival outcome measures. Except for the percentage of participants with EGFR mutation at Screening, outcome measures were not analyzed for the total collective population.
    End point values
    Erlotinib Untreated
    Number of subjects analysed
    3
    21
    Units: percentage of participants
        At 6 months
    100
    67
        At 12 months
    100
    24
    No statistical analyses for this end point

    Secondary: Percentage of Participants with EGFR Mutation at Screening

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    End point title
    Percentage of Participants with EGFR Mutation at Screening [6]
    End point description
    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. All erlotinib-treated participants who received at least one dose of erlotinib, in addition to all enrolled untreated patients, were included in the analysis.
    End point type
    Secondary
    End point timeframe
    Screening
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Erlotinib-treated participants were analyzed for all reported outcome measures; untreated participants were only followed for overall survival and are thus only included in the survival outcome measures. The percentage of participants with EGFR mutation at Screening was reported for the total collective population.
    End point values
    Total Population
    Number of subjects analysed
    24
    Units: percentage of participants
    17
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 2 years
    Adverse event reporting additional description
    Only participants treated with erlotinib were assessed for adverse events.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    NCI CTC-AE
    Dictionary version
    4.0
    Reporting groups
    Reporting group title
    Erlotinib
    Reporting group description
    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.

    Serious adverse events
    Erlotinib
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 3 (33.33%)
         number of deaths (all causes)
    2
         number of deaths resulting from adverse events
    0
    Infections and infestations
    Epidural catheter infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Erlotinib
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lipoma
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Periosteal reaction
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nervous system disorders
    Paraparesis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Bleeding from mouth
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Infections and infestations
    Eye infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Sep 2011
    The study design was modified to collect follow-up information on survival in each study arm.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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