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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of BG00011 in Patients With Idiopathic Pulmonary Fibrosis

    Summary
    EudraCT number
    2017-003158-18
    Trial protocol
    CZ   GB   DK   NL   GR   BE   ES   DE   PL   IT  
    Global end of trial date
    14 Nov 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Nov 2020
    First version publication date
    14 Nov 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    203PF203
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03573505
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Biogen
    Sponsor organisation address
    250 Binney Street, Cambridge, United States, 02142
    Public contact
    Biogen Study Medical Director, Biogen, clinicaltrials@biogen.com
    Scientific contact
    Biogen Study Medical Director, Biogen, clinicaltrials@biogen.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
    14 Nov 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Nov 2019
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate the efficacy of BG00011 compared with placebo in subjects with Idiopathic Pulmonary Fibrosis (IPF).
    Protection of trial subjects
    Written informed consent was obtained from each subject or subject’s legally authorized representative (e.g., parent or legal guardian), as applicable, prior to evaluations performed for eligibility. Subjects or the subject’s legally authorized representative were given adequate time to review the information in the informed consent/assent and were allowed to ask, and have answered, questions concerning all portions of the conduct of the study.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    24 Sep 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety, Efficacy
    Long term follow-up duration
    2 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 29
    Country: Number of subjects enrolled
    Australia: 28
    Country: Number of subjects enrolled
    Netherlands: 8
    Country: Number of subjects enrolled
    Korea, Republic of: 7
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    Chile: 6
    Country: Number of subjects enrolled
    Czech Republic: 6
    Country: Number of subjects enrolled
    Greece: 3
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    Argentina: 2
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    Poland: 2
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Israel: 1
    Country: Number of subjects enrolled
    Russian Federation: 1
    Country: Number of subjects enrolled
    Spain: 1
    Worldwide total number of subjects
    106
    EEA total number of subjects
    32
    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)
    30
    From 65 to 84 years
    76
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Subjects were enrolled at 47 investigational sites in 16 countries from September 24, 2018 to November 14, 2019. Out of 109 enrolled subjects, 106 were treated (mITT population) and 3 were not treated. Data for subject number per country and age-group breakdown is provided for 106 subjects as data for age for one of the subjects was not collected.

    Pre-assignment
    Screening details
    A total of 109 subjects with Idiopathic pulmonary fibrosis (IPF) were enrolled and randomized in this study. Of which, 106 subjects received at least one dose of study drug (mITT). The maximum length of the dosing period was up to Week 46. Subjects were then followed-up for safety for up to Week 60.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Subjects received BG00011-matching placebo, once weekly by subcutaneous (SC) injection up to a maximum of 46 weeks. Median exposure to placebo was for 19.14 weeks.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subcutaneous (SC) injection, once weekly.

    Arm title
    BG00011
    Arm description
    Subjects received BG00011 56 milligram (mg), once weekly by SC injection up to a maximum of 46 weeks. Median exposure to BG00011 was for 17.14 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    BG00011
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    SC injection, once weekly.

    Number of subjects in period 1
    Placebo BG00011
    Started
    52
    54
    Completed
    0
    0
    Not completed
    52
    54
         Death
    -
    4
         Consent withdrawn
    -
    1
         Study terminated by sponsor
    50
    45
         Disease progression
    -
    1
         Site Terminated by Sponsor
    1
    1
         Adverse Event
    1
    1
         Reason not specified
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Subjects received BG00011-matching placebo, once weekly by subcutaneous (SC) injection up to a maximum of 46 weeks. Median exposure to placebo was for 19.14 weeks.

    Reporting group title
    BG00011
    Reporting group description
    Subjects received BG00011 56 milligram (mg), once weekly by SC injection up to a maximum of 46 weeks. Median exposure to BG00011 was for 17.14 weeks.

    Reporting group values
    Placebo BG00011 Total
    Number of subjects
    52 54 106
    Age categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    68.50 ± 6.652 69.46 ± 7.570 -
    Sex: Female, Male
    Units: subjects
        Female
    12 11 23
        Male
    40 43 83
    Race/Ethnicity Customized
    Units: Subjects
        Hispanic or Latino
    6 5 11
        Not Hispanic or Latino
    46 49 95
    Race/Ethnicity Customized
    Units: Subjects
        Asian
    2 5 7
        Black or African American
    0 1 1
        White
    49 47 96
        Other
    1 1 2

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Subjects received BG00011-matching placebo, once weekly by subcutaneous (SC) injection up to a maximum of 46 weeks. Median exposure to placebo was for 19.14 weeks.

    Reporting group title
    BG00011
    Reporting group description
    Subjects received BG00011 56 milligram (mg), once weekly by SC injection up to a maximum of 46 weeks. Median exposure to BG00011 was for 17.14 weeks.

    Primary: Change from Baseline in Forced (Expiratory) Vital Capacity (FVC) at Week 52

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    End point title
    Change from Baseline in Forced (Expiratory) Vital Capacity (FVC) at Week 52 [1]
    End point description
    FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry and is the most important measurement of lung function. This test requires subject to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint in this endpoint. 'n' at Week 52, are a few subjects whose early termination visit fell into the analysis visit window of the Week 52 visit. No subject received Week 52 dosing.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    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 for this endpoint.
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: litres (L)
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    2.883 ± 0.7037
    2.867 ± 0.8607
        Change at Week 52 (n=5,4)
    -0.308 ± 0.1768
    -0.455 ± 0.2849
    No statistical analyses for this end point

    Secondary: Change from Baseline in FVC, Expressed in Percent Predicted at Week 52

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    End point title
    Change from Baseline in FVC, Expressed in Percent Predicted at Week 52
    End point description
    FVC is the total amount of air exhaled during the forced expiratory volume test measured during spirometry and is the most important measurement of lung function. It requires subject to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate subject's bronchial (breathing) tubes. Percent predicted FVC (in %, here FVC was measured in litres) = [(observed FVC)/(predicted FVC)]*100. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint. 'n' at Week 52, are a few subjects whose early termination visit fell into the analysis visit window of the Week 52 visit. No subject received Week 52 dosing.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: percentage of of predicted FVC
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    76.1 ± 15.46
    77.4 ± 17.07
        Change at Week 52 (n=5,4)
    -7.6 ± 5.22
    -11.5 ± 6.95
    No statistical analyses for this end point

    Secondary: Time to Progression

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    End point title
    Time to Progression
    End point description
    Time to progression is defined by a composite endpoint, including any of the following events: Absolute decline of 10% predicted in FVC (FVC percent predicted (baseline) - FVC percent predicted (progression) ≥10%); Non-elective hospitalization for respiratory events; Lung transplantation or death. The earliest time to meet at least 1 composite criterion was calculated. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'Number of subjects analysed' are the subjects who were assessed in this outcome measure.
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    8
    15
    Units: days
        median (full range (min-max))
    127.5 (29 to 235)
    119.0 (28 to 302)
    Statistical analysis title
    Placebo vs BG00011
    Statistical analysis description
    A cox proportional hazards model with terms for treatment (BG00011 vs. placebo) and randomization stratification factor is used. A stratified log-rank test is used to compare the 2 treatment groups using randomization stratus as the stratification factor. An HR (Hazard Ratio) < 1 indicates lower risk of event for the BG00011 group where HR is based on Cox proportional hazard model with treatment (Placebo, BG00011) as the categorical covariate.
    Comparison groups
    Placebo v BG00011
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.112
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    2.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.85
         upper limit
    4.73

    Secondary: Time to First Acute Idiopathic Pulmonary Fibrosis (IPF) Exacerbation

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    End point title
    Time to First Acute Idiopathic Pulmonary Fibrosis (IPF) Exacerbation
    End point description
    Time to first acute IPF exacerbation is defined as time from randomization to the first occurrence of acute IPF exacerbation. Acute IPF exacerbation is defined as a clinically significant deterioration of unidentifiable cause in a subject with underlying IPF. The diagnostic criteria for IPF used in this study were derived from evidence-based guidelines developed by the American Thoracic Society/European Respiratory Society/Japanese Respiratory Society/Latin American Thoracic Association (ATS/ERS/JRS/ALAT) joint task force for the diagnosis and management of IPF. Participants were assessed according to the modified 2007 diagnostic criteria for acute IPF exacerbation. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'Number of subjects analysed' are the subjects who had at least one acute IPF exacerbation.
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    0 [2]
    7
    Units: days
        median (full range (min-max))
    ( to )
    114.0 (42 to 223)
    Notes
    [2] - No subjects had at least one acute IPF exacerbation in Placebo arm.
    No statistical analyses for this end point

    Secondary: Number of Subjects with at Least One Acute IPF Exacerbation

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    End point title
    Number of Subjects with at Least One Acute IPF Exacerbation
    End point description
    Acute IPF exacerbation is defined as a clinically significant deterioration of unidentifiable cause in a subject with underlying IPF. The diagnostic criteria for IPF used in this study were derived from evidence-based guidelines developed by the American Thoracic Society/European Respiratory Society/Japanese Respiratory Society/Latin American Thoracic Association (ATS/ERS/JRS/ALAT) joint task force for the diagnosis and management of IPF. Participants were assessed according to the modified 2007 diagnostic criteria for acute IPF exacerbation. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo).
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: subjects
    0
    7
    No statistical analyses for this end point

    Secondary: Number of IPF Exacerbations

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    End point title
    Number of IPF Exacerbations
    End point description
    The IPF exacerbation is defined as a clinically significant deterioration of unidentifiable cause in a subject with underlying IPF. The diagnostic criteria for IPF used in this study were derived from evidence-based guidelines developed by the American Thoracic Society/European Respiratory Society/Japanese Respiratory Society/Latin American Thoracic Association (ATS/ERS/JRS/ALAT) joint task force for the diagnosis and management of IPF. Participants were assessed according to the modified 2007 diagnostic criteria for acute IPF exacerbation. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo).
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: exacerbations
    0
    8
    No statistical analyses for this end point

    Secondary: Number of Subjects with Absolute Decline of 10% Predicted in FVC

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    End point title
    Number of Subjects with Absolute Decline of 10% Predicted in FVC
    End point description
    FVC is the is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry and is the most important measurement of lung function. This test requires subject to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate subject's bronchial (breathing) tubes. Absolute Decline of 10% = FVC percent predicted (baseline) - FVC percent predicted (progression) ≥10%. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo).
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: subjects
    6
    9
    No statistical analyses for this end point

    Secondary: Time to Death or Lung Transplantation

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    End point title
    Time to Death or Lung Transplantation
    End point description
    Time to Death or Lung Transplantation is defined as the time from randomization to the first occurrence of any one of the event (death or lung transplantation). MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or Placebo).
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    0 [3]
    54
    Units: days
    median (full range (min-max))
        Time to Death
    ( to )
    83.0 (60 to 137)
        Time to Lung Transplantation
    ( to )
    155.0 (155 to 155)
    Notes
    [3] - No subjects were analysed for this endpoint in the Placebo arm.
    No statistical analyses for this end point

    Secondary: Time to All Non-elective Hospitalizations

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    End point title
    Time to All Non-elective Hospitalizations
    End point description
    Time to all non-elective hospitalizations is defined as the time from randomization to the first occurrence of hospitalization which was not elected by the participant. The MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or Placebo). 'Number of subjects analysed' are the subjects who had at least one episode of non-elective hospitalization.
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    5
    11
    Units: days
        median (full range (min-max))
    133.0 (61 to 235)
    119.0 (42 to 302)
    No statistical analyses for this end point

    Secondary: Time to All Non-Elective Respiratory Hospitalizations

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    End point title
    Time to All Non-Elective Respiratory Hospitalizations
    End point description
    Time to all non-elective respiratory hospitalizations is defined as the time from randomization to the first occurrence of hospitalization due to respiratory problems, which was not elected by the participant. The MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or Placebo). 'Number of subjects analysed' are the subjects who had at least one episode of non-elective respiratory hospitalization.
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    2
    9
    Units: days
        median (full range (min-max))
    205.0 (175 to 235)
    119.0 (42 to 223)
    No statistical analyses for this end point

    Secondary: Change from Baseline in Absolute FVC

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    End point title
    Change from Baseline in Absolute FVC
    End point description
    FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry and is the most important measurement of lung function. This test requires subject to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate subject's bronchial (breathing) tubes. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). ‘n’ are the subjects who were assessed at the specified timepoint in this endpoint.
    End point type
    Secondary
    End point timeframe
    Up to Week 44
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: litres
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    2.883 ± 0.7037
    2.867 ± 0.8607
        Change at Week 4 (n=51,52)
    0.006 ± 0.1544
    0.032 ± 0.1526
        Change at Week 8 (n=45,45)
    -0.030 ± 0.1575
    0.007 ± 0.1513
        Change at Week 12 (n=41,39)
    -0.020 ± 0.1846
    0.000 ± 0.2247
        Change at Week 16 (n=37,39)
    -0.046 ± 0.1819
    -0.042 ± 0.2400
        Change at Week 20 (n=30,31)
    -0.086 ± 0.1921
    -0.121 ± 0.3318
        Change at Week 26 (n=23,20)
    -0.079 ± 0.1949
    -0.069 ± 0.3045
        Change at Week 32 (n=17,13)
    -0.078 ± 0.1979
    -0.152 ± 0.3142
        Change at Week 38 (n=12,11)
    -0.131 ± 0.1847
    -0.275 ± 0.3436
        Change at Week 44 (n=8,4)
    -0.200 ± 0.2153
    -0.485 ± 0.3816
    No statistical analyses for this end point

    Secondary: Change from Baseline in Percent Predicted FVC

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    End point title
    Change from Baseline in Percent Predicted FVC
    End point description
    FVC is the total amount of air exhaled during the forced expiratory volume test that is measured during spirometry and is the most important measurement of lung function. This test requires subject to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which is used to dilate subject's bronchial (breathing) tubes. Percent predicted FVC (in %) = [(observed FVC)/(predicted FVC)]*100. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint in this endpoint.
    End point type
    Secondary
    End point timeframe
    Up to Week 44
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: percentage of predicted FVC
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    76.1 ± 15.46
    77.4 ± 17.07
        Change at Week 4 (n=51,52)
    0.1 ± 4.13
    1.1 ± 4.52
        Change at Week 8 (n=45,45)
    -0.7 ± 4.22
    0.4 ± 4.12
        Change at Week 12 (n=41,39)
    -0.4 ± 5.31
    0.1 ± 5.79
        Change at Week 16 (n=37,39)
    -1.1 ± 4.97
    -0.8 ± 6.24
        Change at Week 20 (n=30,31)
    -2.3 ± 5.04
    -3.0 ± 8.08
        Change at Week 26 (n=23,20)
    -2.0 ± 5.60
    -1.6 ± 8.19
        Change at Week 32 (n=17,13)
    -1.9 ± 5.44
    -3.7 ± 8.66
        Change at Week 38 (n=12,11)
    -3.4 ± 5.43
    -7.4 ± 8.44
        Change at Week 44 (n=8,4)
    -5.1 ± 6.08
    -12.5 ± 9.26
    No statistical analyses for this end point

    Secondary: Change from Baseline in Absolute Carbon Monoxide Diffusion Capacity (DLco)

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    End point title
    Change from Baseline in Absolute Carbon Monoxide Diffusion Capacity (DLco)
    End point description
    DLCO is a measurement of the ability of the lungs to transfer gases from the air to the blood. Evaluation of DLco was be performed by single-breath carbon monoxide diffusing capacity. DLCO was assessed in milliliters per minute per millimeter of mercury (mL/min/mmHg). Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint in this endpoint.
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: mL/min/mmHg
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    4.397 ± 1.1853
    4.036 ± 1.1043
        Change at Week 4 (n=50,52)
    -0.094 ± 0.4783
    0.019 ± 0.4431
        Change at Week 8 (n=46,43)
    -0.108 ± 0.5583
    0.028 ± 0.6078
        Change at Week 16 (n=34,38)
    -0.205 ± 0.6579
    -0.263 ± 0.5472
        Change at Week 26 (n=23,20)
    -0.074 ± 0.7756
    -0.383 ± 0.7427
        Change at Week 38 (n=11,11)
    -0.025 ± 0.5611
    -0.445 ± 0.6153
        Change at Week 52 (n=5,4)
    -0.228 ± 0.1839
    -0.400 ± 0.7921
    No statistical analyses for this end point

    Secondary: Change from Baseline in Percent Predicted Carbon Monoxide Diffusion Capacity (DLco)

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    End point title
    Change from Baseline in Percent Predicted Carbon Monoxide Diffusion Capacity (DLco)
    End point description
    DLCO is a measurement of the ability of the lungs to transfer gases from the air to the blood. Evaluation of DLco was be performed by single-breath carbon monoxide diffusing capacity. Percent of predicted DLco (in %) = [(observed DLco)/(predicted DLco)]*100. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint in this endpoint.
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: percentage of predicted DLco
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    52.6 ± 13.56
    49.1 ± 11.15
        Change at Week 4 (n=50,52)
    -1.1 ± 5.62
    0.4 ± 5.08
        Change at Week 8 (n=46,43)
    -1.5 ± 6.75
    0.5 ± 7.78
        Change at Week 16 (n=34,38)
    -2.4 ± 7.48
    -3.1 ± 6.49
        Change at Week 26 (n=23,20)
    -1.3 ± 9.10
    -4.8 ± 8.78
        Change at Week 38 (n=11,11)
    -0.5 ± 6.42
    -5.5 ± 6.82
        Change at Week 52 (n=5,4)
    -2.4 ± 1.82
    -4.0 ± 9.56
    No statistical analyses for this end point

    Secondary: Change from Baseline in Absolute Total Lung Capacity (TLC)

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    End point title
    Change from Baseline in Absolute Total Lung Capacity (TLC)
    End point description
    Total lung capacity is the measure of lung volume was measured by full-body plethysmography. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified timepoint in this endpoint. 9999 indicates that the data was not reported as no subjects were evaluated at the given time point.
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: litres
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    4.472 ± 1.0497
    4.413 ± 1.0560
        Change at Week 4 (n=1,4)
    0.090 ± 99999
    -0.080 ± 0.2276
        Change at Week 8 (n=1,0)
    -0.010 ± 99999
    9999 ± 9999
        Change at Week 12 (n=3,1)
    -0.113 ± 0.3272
    0.380 ± 99999
        Change at Week 16 (n=1,4)
    0.060 ± 99999
    -0.350 ± 0.2131
        Change at Week 20 (n=5,5)
    -0.002 ± 0.2344
    -0.310 ± 0.3476
        Change at Week 26 (n=9,9)
    -0.103 ± 0.2293
    -0.214 ± 0.4403
        Change at Week 32 (n=1,1)
    -0.370 ± 99999
    0.140 ± 99999
        Change at Week 38 (n=2,3)
    -0.090 ± 0.2263
    -0.023 ± 0.4754
        Change at Week 44 (n=2,0)
    -0.160 ± 0.0283
    9999 ± 9999
        Change at Week 52 (n=3,2)
    -0.197 ± 0.3075
    -0.695 ± 0.3465
    No statistical analyses for this end point

    Secondary: Change from Baseline in Percent Predicted TLC

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    End point title
    Change from Baseline in Percent Predicted TLC
    End point description
    Total lung capacity is the measure of lung volume was measured by full-body plethysmography. Percent of predicted TLC (in %) = [(observed TLC)/(predicted TLC)]*100. Change from baseline is defined as post-baseline value minus baseline value. A negative change from baseline indicates decline. MITT population included all participants who were randomized and received at least 1 dose of study treatment (BG00011 or placebo). 'n’ are the subjects who were assessed at the specified time point in this endpoint. 99999 indicates that SD was not calculable as there was only 1 subject. 9999 indicates that the data was not reported as no subjects were evaluated at the given time point.
    End point type
    Secondary
    End point timeframe
    Up to Early Termination Visit (Up to Week 52)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: percentage of predicted TLC
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    69.6 ± 14.67
    67.7 ± 12.74
        Change at Week 4 (n=1,4)
    2.0 ± 99999
    -1.3 ± 3.50
        Change at Week 8 (n=1,0)
    0.0 ± 99999
    9999 ± 9999
        Change at Week 12 (n=3,1)
    -1.3 ± 4.93
    5.0 ± 99999
        Change at Week 16 (n=1,4)
    1.0 ± 99999
    -4.8 ± 2.75
        Change at Week 20 (n=5,5)
    0.0 ± 3.81
    -5.8 ± 5.76
        Change at Week 26 (n=9,9)
    -1.6 ± 3.78
    -3.0 ± 6.12
        Change at Week 32 (n=1,1)
    -5.0 ± 99999
    3.0 ± 99999
        Change at Week 38 (n=2,3)
    -2.0 ± 4.24
    -0.3 ± 6.51
        Change at Week 44 (n=2,0)
    -2.0 ± 0.00
    9999 ± 9999
        Change at Week 52 (n=3,2)
    -2.7 ± 3.79
    -10.5 ± 3.54
    No statistical analyses for this end point

    Secondary: Change from Baseline in 6 Minute Walk Test (6MWT) Parameters

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    End point title
    Change from Baseline in 6 Minute Walk Test (6MWT) Parameters
    End point description
    The 6MWT measures the distance (in meters), a subject is able to walk in 6 minutes. This test measures the distance a person can walk quickly on a flat, hard surface in 6 minutes and reflects an individual's ability to perform daily physical activities. MITT population included all subjects who were randomised and received at least 1 dose of study treatment (BG00011 or placebo). 'n' are subjects with data available for analyses at given timepoint. Subjects at Week 52, are a few subjects whose early termination visit fell into the analysis visit window of the Week 52 visit. No subjects received Week 52 dosing.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26 and Week 52
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: meters
    arithmetic mean (standard deviation)
        Baseline (n=52,54)
    458.4 ± 115.33
    404.0 ± 122.61
        Change at Week 26 (n=24,20)
    -5.9 ± 38.79
    -28.6 ± 67.21
        Change at Week 52 (n=4,4)
    44.8 ± 87.16
    -40.0 ± 94.60
    No statistical analyses for this end point

    Secondary: Number of Subjects with Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    Number of Subjects with Adverse Events (AEs) and Serious Adverse Events (SAEs)
    End point description
    An AE is any untoward medical occurrence in a subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, requires inpatient hospitalization, results in persistent or significant disability and/or results in a congenital anomaly. The safety population included all subjects who were randomised and receive at least 1 dose of study treatment (BG00011 or placebo).
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    52
    54
    Units: subjects
        AEs
    39
    47
        SAEs
    7
    15
    No statistical analyses for this end point

    Secondary: Number of Subjects With Anti-BG00011 Antibodies in the Serum

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    End point title
    Number of Subjects With Anti-BG00011 Antibodies in the Serum
    End point description
    The immunogenicity population defined as subjects from mITT population who have at least 1 postdose immunogenicity sample evaluated for anti-BG00011 antibodies. 'n' are the subjects who were assessed in this endpoint.
    End point type
    Secondary
    End point timeframe
    Up to Week 60 (End of Study)
    End point values
    Placebo BG00011
    Number of subjects analysed
    46
    42
    Units: subjects
    1
    0
    No statistical analyses for this end point

    Secondary: Concentration of BG00011 in the Serum

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    End point title
    Concentration of BG00011 in the Serum [4]
    End point description
    Pharmacokinetics (PK) population included all subjects who received at least 1 dose of study treatment and had at least one PK concentration measurement. "n" indicates number of subjects analysed at the give time point. Subjects at Week 52 are a few subjects whose early termination visit fell into the analysis visit window of the Week 52 visit. No participant received Week 52 dosing.
    End point type
    Secondary
    End point timeframe
    Predose on Day 0, Day 5, Week 4, Week 8, Week 12, Week 26, Week 38, Week 52, and Safety Follow-up Visit (Up to Week 60).
    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: The serum concentration of BG00011 is measured in subjects in BG00011 arm.
    End point values
    BG00011
    Number of subjects analysed
    54
    Units: nanograms (ng)/mL
    arithmetic mean (standard deviation)
        Baseline (n=54)
    0 ± 0
        Day 5 (n=53)
    2292.83 ± 1778.376
        Week 4 (n=53)
    5323.08 ± 3058.436
        Week 8 (n=44)
    7971.05 ± 4912.091
        Week 12 (n=39)
    7768.33 ± 5139.179
        Week 16 (n=37)
    6934.86 ± 4288.642
        Week 20 (n=29)
    6614.24 ± 4203.020
        Week 26 (n=19)
    8579.42 ± 9754.642
        Week 38 (n=10)
    3560.00 ± 2771.630
        Week 52 (n=3)
    1085.33 ± 455.978
        Safety follow-up (n=9)
    344.64 ± 701.639
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 60 Weeks (End of Study)
    Adverse event reporting additional description
    The safety population included all the subjects who were randomised and receive at least 1 dose of study treatment (BG00011 or placebo).
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    -

    Reporting group title
    BG00011
    Reporting group description
    -

    Serious adverse events
    Placebo BG00011
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 52 (13.46%)
    15 / 54 (27.78%)
         number of deaths (all causes)
    0
    4
         number of deaths resulting from adverse events
    Vascular disorders
    Steal syndrome
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Joint dislocation
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ovarian adenoma
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Prostate cancer
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Dyspnoea
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Idiopathic pulmonary fibrosis
         subjects affected / exposed
    0 / 52 (0.00%)
    8 / 54 (14.81%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 9
         deaths causally related to treatment / all
    0 / 0
    0 / 2
    Pneumothorax spontaneous
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Carotid artery stenosis
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cerebrovascular accident
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diverticulum
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Fallopian tube cyst
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ovarian cyst
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hip deformity
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 54 (3.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Urinary tract infection
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 54 (3.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo BG00011
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    31 / 52 (59.62%)
    40 / 54 (74.07%)
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    0 / 52 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    3
    Fall
         subjects affected / exposed
    1 / 52 (1.92%)
    4 / 54 (7.41%)
         occurrences all number
    1
    4
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    10 / 52 (19.23%)
    9 / 54 (16.67%)
         occurrences all number
    11
    10
    Cough
         subjects affected / exposed
    15 / 52 (28.85%)
    11 / 54 (20.37%)
         occurrences all number
    17
    14
    Epistaxis
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 54 (5.56%)
         occurrences all number
    1
    8
    Idiopathic pulmonary fibrosis
         subjects affected / exposed
    0 / 52 (0.00%)
    6 / 54 (11.11%)
         occurrences all number
    0
    7
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 52 (9.62%)
    1 / 54 (1.85%)
         occurrences all number
    6
    1
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 52 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    4
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    8 / 52 (15.38%)
    4 / 54 (7.41%)
         occurrences all number
    8
    4
    Injection site bruising
         subjects affected / exposed
    4 / 52 (7.69%)
    2 / 54 (3.70%)
         occurrences all number
    4
    2
    Injection site pain
         subjects affected / exposed
    0 / 52 (0.00%)
    4 / 54 (7.41%)
         occurrences all number
    0
    4
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    5 / 52 (9.62%)
    13 / 54 (24.07%)
         occurrences all number
    7
    14
    Nausea
         subjects affected / exposed
    5 / 52 (9.62%)
    5 / 54 (9.26%)
         occurrences all number
    8
    6
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 54 (0.00%)
         occurrences all number
    4
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 52 (5.77%)
    3 / 54 (5.56%)
         occurrences all number
    3
    3
    Back pain
         subjects affected / exposed
    2 / 52 (3.85%)
    3 / 54 (5.56%)
         occurrences all number
    2
    4
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 52 (0.00%)
    3 / 54 (5.56%)
         occurrences all number
    0
    3
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 54 (5.56%)
         occurrences all number
    1
    3
    Nasopharyngitis
         subjects affected / exposed
    3 / 52 (5.77%)
    7 / 54 (12.96%)
         occurrences all number
    4
    8
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 52 (11.54%)
    5 / 54 (9.26%)
         occurrences all number
    7
    7

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Jul 2019
    The primary reason for this amendment to Protocol 203PF203 is to reflect current modified (2018) American Thoracic Society (ATS) guidelines for idiopathic pulmonary fibrosis (IPF) diagnosis. • The stratification of subjects on background therapy was clarified. • Text was added to clarify that the Investigator should attempt to complete the Investigator’s determination of IPF exacerbation as soon as possible. • The exclusion criteria due to current hepatitis C or hepatitis B infection was clarified. • Liver chemistry threshold discontinuation criteria were added. • Dosing can be performed 2 days from the dose schedule. • A subject may continue in the study on their assigned study treatment if background therapy is initiated or adjusted. • The interim analysis when approximately 50% of subjects completed the week 52 visit was removed. A potential interim database lock and its associated interim analysis for efficacy and safety data is added.

    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.
    The study was terminated early due to safety findings
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
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