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    Clinical Trial Results:
    A Phase 2, Multi-Centre, Open-Label, Single-Arm Trial Investigating the Safety, Efficacy and Pharmacokinetics of C21 in Subjects with Idiopathic Pulmonary Fibrosis

    Summary
    EudraCT number
    		 2020-000822-24
    Trial protocol
    		 GB  
    Global end of trial date
    30 Mar 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 Mar 2025
    First version publication date
    19 Mar 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VP-C21-005
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04533022
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vicore Pharma AB
    Sponsor organisation address
    Kornhamnstorg 53, Stockholm, Sweden, SE-111 27
    Public contact
    Anne Katrine Cohrt, Vicore Pharma AB, anne-katrine.cohrt@vicorepharma.com
    Scientific contact
    Bertil Lindmark, Vicore Pharma AB, bertil.lindmark@vicorepharma.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
    10 Jun 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Mar 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Mar 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the safety of C21 with 200 mg daily dose (100 mg b.i.d.) administered orally to subjects with IPF.
    Protection of trial subjects
    None.
    Background therapy
    		 None
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Nov 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    India: 38
    Country: Number of subjects enrolled
    Ukraine: 4
    Country: Number of subjects enrolled
    Russian Federation: 3
    Worldwide total number of subjects
    52
    EEA total number of subjects
    0
    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)
    18
    From 65 to 84 years
    34
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 One hundred and thirty-eight subjects provided informed consent and were enrolled in the trial; 86 of these subjects were screening failures. The remaining 52 subjects received at least one dose of IMP.

    Period 1
    Period 1 title
    Trial period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 C21 100 mg BID
    Arm description
    		 Oral capsules of C21 (buloxibutid) 100 mg administered twice daily (BID)
    Arm type
    		 Experimental

    Investigational medicinal product name
    C21
    Investigational medicinal product code
    Other name
    compound 21, buloxibutid
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    100 mg twice daily (BID) for 36 weeks

    Number of subjects in period 1
    		C21 100 mg BID
    Started
    52
    Completed
    27
    Not completed
    25
         Adverse event, serious fatal
    2
         Consent withdrawn by subject
    15
         FVC decline, FVCpp<60% and worsening of resp symp
    1
         Adverse event, non-fatal
    5
         FVC decline and FVCpp<60%
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Trial period
    Reporting group description
    		 -

    Reporting group values
    		 Trial period Total
    Number of subjects
    		 52 52
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 18 18
        From 65-84 years
    		 34 34
        85 years and over
    		 0 0
    Age continuous
    Age at screening
    Units: years
        arithmetic mean (standard deviation)
    		 67.3 ( 9.38 ) -
    Gender categorical
    Units: Subjects
        Female
    		 12 12
        Male
    		 40 40
    Ethnic origin
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 52 52
    Race
    Units: Subjects
        Black or African American
    		 0 0
        American Indian or Alaska Native
    		 0 0
        Asian
    		 38 38
        Native Hawaiian or Other Pacific Islander
    		 0 0
        White
    		 14 14
        Other
    		 0 0
    Smoking status
    Units: Subjects
        Current
    		 0 0
        Former
    		 10 10
        Never
    		 42 42
    E-cigarettes and vapes status
    Units: Subjects
        Current
    		 0 0
        Former
    		 1 1
        Never
    		 51 51
    Previous use of antifibrotics
    Units: Subjects
        Nintedanib
    		 0 0
        Pirfenidone
    		 0 0
        Neither nintedanib or pirfenidone
    		 52 52
    HRCT pattern (central reading)
    HRCT pattern (central reading)
    Units: Subjects
        Typical UIP HRCT Pattern
    		 20 20
        Probable UIP HRCT
    		 32 32
    Height
    Height at screening
    Units: cm
        arithmetic mean (standard deviation)
    		 161.7 ( 8.7 ) -
    Weight
    Weight at screening
    Units: kg
        arithmetic mean (standard deviation)
    		 64.6 ( 14.2 ) -
    BMI
    Body mass index (BMI) at screening
    Units: kg/m^2
        arithmetic mean (standard deviation)
    		 24.57 ( 4.12 ) -
    Time since diagnosis of IPF
    Units: Years
        arithmetic mean (standard deviation)
    		 1.01 ( 1.195 ) -
    Oxygen saturation at screening
    Units: percent
        arithmetic mean (standard deviation)
    		 95.3 ( 2.40 ) -
    FEV1
    Units: liters
        arithmetic mean (standard deviation)
    		 1.912 ( 0.528 ) -
    FVC
    Units: Liters
        arithmetic mean (standard deviation)
    		 2.387 ( 0.667 ) -
    FEV1 (% predicted normal)
    Units: Percent
        arithmetic mean (standard deviation)
    		 77.51 ( 14.687 ) -
    FVC (% predicted normal)
    Units: percent
        arithmetic mean (standard deviation)
    		 75.46 ( 13.662 ) -
    FEV1/FVC (ratio)
    Units: Ratio
        arithmetic mean (standard deviation)
    		 0.804 ( 0.0673 ) -
    Age of HRCT scans
    Units: Months
        arithmetic mean (standard deviation)
    		 5.58 ( 8.720 ) -

    End points

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    End points reporting groups
    Reporting group title
    C21 100 mg BID
    Reporting group description
    		 Oral capsules of C21 (buloxibutid) 100 mg administered twice daily (BID)

    Primary: Nature and frequency of adverse events occurring over the trial period

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    End point title
    		 Nature and frequency of adverse events occurring over the trial period [1]
    End point description
    Adverse events were recorded from signing of informed consent until end of trial. Nature and frequency of adverse events are presented in the adverse events section.
    End point type
    Primary
    End point timeframe
    From signing of informed consent until end of trial.
    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: A statistical analysis has not been made on the safety data. More details on the safety data are provided in the Adverse event section.
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    52
    Units: Subjects
        Total number of subjects with TEAEs
    37
        Total number of subjects with serious TEAEs
    5
        Subjects with serious treatment-related TEAEs
    0
        Total number of subjects with TEAEs leading to wi
    6
        Total number of subjects with TEAEs leading to di
    12
        Total number of subj. w. TEAEs leading to death
    2
        Subjects with treatment-rel. TEAEs lead to disc.
    3
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity (Non-imputed Data)

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    End point title
    		 Change From Baseline in Forced Vital Capacity (Non-imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    35
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -4.9 (-73.2 to 63.4)
        Week 24
    16.0 (-117.4 to 149.3)
        Week 36
    216.0 (36.9 to 395.1)
    No statistical analyses for this end point

    Secondary: Change from baseline in forced vital capacity (imputed data)

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    End point title
    		 Change from baseline in forced vital capacity (imputed data)
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    48
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -57.6 (-123.3 to 8.1)
        Week 24
    -70.3 (-170.5 to 29.9)
        Week 36
    9.4 (-115.6 to 134.4)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity - FVC12AS (Imputed Data)

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    End point title
    		 Change From Baseline in Forced Vital Capacity - FVC12AS (Imputed Data)
    End point description
    Mean changes in force vital capacity (mL) from baseline to Week 12, 24, and 36 were calculated for the FVC Week 12 analysis set (FVC12AS) (imputed data). FVC12AS is the subset of subjects that had not withdrawn at Week 12.
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    37
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -6.6 (-71.3 to 58.0)
        Week 24
    -5.2 (-121.1 to 110.7)
        Week 36
    116.0 (-27.4 to 259.3)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity - FVC24AS (Imputed Data)

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    End point title
    		 Change From Baseline in Forced Vital Capacity - FVC24AS (Imputed Data)
    End point description
    Mean changes in force vital capacity (mL) from baseline to Week 12, 24, and 36 were calculated for the FVC Week 24 analysis set (FVC24AS) (imputed data). FVC24AS is the subset of subjects who had not withdrawn at Week 24.
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    32
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    0.6 (-67.2 to 68.3)
        Week 24
    16.0 (-117.4 to 149.3)
        Week 36
    165.5 (4.1 to 326.9)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity - PPAS (Imputed Data)

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    End point title
    		 Change From Baseline in Forced Vital Capacity - PPAS (Imputed Data)
    End point description
    Mean changes in force vital capacity (mL) from baseline to Week 12, 24, and 36 were calculated in the per protocol analysis set (PPAS)
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    45
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -51.5 (-115.7 to 12.7)
        Week 24
    -53.2 (-158.2 to 51.9)
        Week 36
    33.1 (-98.6 to 164.8)
    No statistical analyses for this end point

    Secondary: Change in forced vital capacity by subgroup, Week 12 (imputed data)

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    End point title
    		 Change in forced vital capacity by subgroup, Week 12 (imputed data)
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    48
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    -107.9 (-241.3 to 25.4)
        Probable UIP
    -27.5 (-100.1 to 45.1)
        India
    -27.4 (-98.4 to 43.6)
        Rest of world
    -139.0 (-298.0 to 20.0)
        FVC predicted normal ≤ 70%
    4.8 (-78.1 to 87.7)
        FVC predicted normal: > 70%
    -106.2 (-204.8 to -7.7)
    No statistical analyses for this end point

    Secondary: Change in Forced Vital Capacity by Subgroup, Week 24 (Imputed Data)

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    End point title
    		 Change in Forced Vital Capacity by Subgroup, Week 24 (Imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    48
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    -95.8 (-268.9 to 77.3)
        Probable UIP
    -55.0 (-183.8 to 73.7)
        India
    -44.8 (-165.9 to 76.3)
        Rest of world
    -139.1 (-333.5 to 55.4)
        FVC predicted normal ≤ 70%
    -2.8 (-161.2 to 155.6)
        FVC predicted normal: > 70%
    -122.8 (-256.8 to 11.1)
    No statistical analyses for this end point

    Secondary: Change in Forced Vital Capacity by Subgroup, Week 36 (Imputed Data)

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    End point title
    		 Change in Forced Vital Capacity by Subgroup, Week 36 (Imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    48
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    -161.1 (-338.4 to 16.2)
        Probable UIP
    111.6 (-56.4 to 279.6)
        India
    58.3 (-101.6 to 218.3)
        Rest of world
    -122.5 (-300.9 to 55.9)
        FVC predicted normal: ≤70%
    58.1 (-98.4 to 214.6)
        FVC predicted normal: >70%
    -28.6 (-221.4 to 164.3)
    No statistical analyses for this end point

    Secondary: Change in Forced Vital Capacity by Subgroup, Week 12 (non-Imputed Data)

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    End point title
    		 Change in Forced Vital Capacity by Subgroup, Week 12 (non-Imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    35
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    8.3 (-116.1 to 132.6)
        Probable UIP
    -11.7 (-99.0 to 75.6)
        India
    11.9 (-62.3 to 86.0)
        Rest of world
    -53.3 (-235.3 to 128.6)
        FVC predicted normal ≤ 70%
    3.0 (-91.3 to 97.3)
        FVC predicted normal: > 70%
    -13.2 (-121.3 to 94.8)
    No statistical analyses for this end point

    Secondary: Change in Forced Vital Capacity by Subgroup, Week 24 (Non-Imputed Data)

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    End point title
    		 Change in Forced Vital Capacity by Subgroup, Week 24 (Non-Imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    32
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    81.3 (-125.3 to 287.8)
        Probable UIP
    -18.2 (-199.5 to 163.0)
        India
    41.6 (-136.0 to 219.3)
        Rest of world
    -40.5 (-249.2 to 168.2)
        FVC predicted normal ≤ 70%
    40.3 (-181.8 to 262.4)
        FVC predicted normal: > 70%
    -5.5 (-182.0 to 171.1)
    No statistical analyses for this end point

    Secondary: Change in Forced Vital Capacity by Subgroup, Week 36 (Non-Imputed Data)

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    End point title
    		 Change in Forced Vital Capacity by Subgroup, Week 36 (Non-Imputed Data)
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    28
    Units: mL
    arithmetic mean (confidence interval 90%)
        Typical UIP
    27.2 (-216.3 to 270.7)
        Probable UIP
    320.9 (73.0 to 568.8)
        India
    276.5 (31.0 to 521.9)
        Rets of world
    64.9 (-103.7 to 233.5)
        FVC predicted normal ≤ 70%
    239.0 (-2.0 to 480.0)
        FVC predicted normal: > 70%
    198.8 (-77.7 to 475.2)
    No statistical analyses for this end point

    Secondary: Rate of forced vital capacity decline over time, FAS

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    End point title
    		 Rate of forced vital capacity decline over time, FAS
    End point description
    Mean Rates of forced vital capacity decline over time (mL) normalized to change over 24 weeks were calculated using a piece-wise linear regression model (non-imputed data).
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    50
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -8.2 (-141.9 to 125.5)
        Week 24
    36.4 (-87.1 to 160.0)
        Week 36
    108.9 (-4.8 to 222.6)
    No statistical analyses for this end point

    Secondary: Rate of forced vital capacity decline over time, FVC12AS

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    End point title
    		 Rate of forced vital capacity decline over time, FVC12AS
    End point description
    Mean Rates of forced vital capacity decline over time (mL) normalized to change over 24 weeks were calculated in the FVC Week 12 analysis set (FVC12AS) using a Piece-wise Linear Regression Model (non-imputed data). FVC12AS is the subset of subjects that had not withdrawn at Week 12.
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    37
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    25.1 (-140.7 to 190.8)
        Week 24
    26.9 (-105.6 to 159.4)
        Week 36
    113.9 (0.5 to 227.3)
    No statistical analyses for this end point

    Secondary: Rate of forced vital capacity decline over time, FVC24AS

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    End point title
    		 Rate of forced vital capacity decline over time, FVC24AS
    End point description
    Mean Rates of forced vital capacity decline over time (mL) normalized to change over 24 weeks were calculated in the FVC Week 24 analysis set (FVC24AS) using a Piece-wise Linear Regression Model. FVC24AS is the subset of subjects that had not withdrawn at Week 24.
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    32
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    33.8 (-148.5 to 216.0)
        Week 24
    46.4 (-98.6 to 191.3)
        Week 36
    123.5 (7.9 to 239.2)
    No statistical analyses for this end point

    Secondary: Change in Forced expiratory volume in the first second (mL)

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    End point title
    		 Change in Forced expiratory volume in the first second (mL)
    End point description
    Mean changes in FEV1 from baseline to 12, 24, and 36 weeks were calculated.
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    35
    Units: mL
    arithmetic mean (confidence interval 90%)
        Week 12
    -34.1 (-91.3 to 23.2)
        Week 24
    -24.8 (-115.5 to 65.8)
        Week 36
    81.4 (-40.9 to 203.7)
    No statistical analyses for this end point

    Secondary: Plasma concentration of C21 evaluated in a subset of subjects, Day 1

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    End point title
    		 Plasma concentration of C21 evaluated in a subset of subjects, Day 1
    End point description
    Values below the LLOQ have been set to half the LLOQ (LLOQ = 10ng/mL).
    End point type
    Secondary
    End point timeframe
    Day 1
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    12
    Units: ng/mL
    arithmetic mean (standard deviation)
        Prior to dosing
    5.00 ( 0.00 )
        30 min post dose
    1622.00 ( 1453.64 )
        1 h post dose
    1788.11 ( 1112.484 )
        2 h post dose
    864.46 ( 1062.15 )
        3 h post dose
    322.19 ( 407.29 )
        4 h post dose
    282.82 ( 379.31 )
    No statistical analyses for this end point

    Secondary: Plasma Concentration of C21 evaluated in a sub-set of subjects, Week 12

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    End point title
    		 Plasma Concentration of C21 evaluated in a sub-set of subjects, Week 12
    End point description
    Values below the LLOQ have been set to half the LLOQ (LLOQ = 10ng/mL).
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    8
    Units: ng/mL
    arithmetic mean (standard deviation)
        Prior to dosing
    27.13 ( 62.58 )
        30 min post dose
    974.28 ( 1028.90 )
        1 h post dose
    1044.02 ( 1128.30 )
        2 h post dose
    794.02 ( 774.34 )
        3 h post dose
    491.25 ( 820.36 )
        4 h post dose
    101.50 ( 113.96 )
    No statistical analyses for this end point

    Secondary: Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Week 24

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    End point title
    		 Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Week 24
    End point description
    Values below the LLOQ have been set to half the LLOQ (LLOQ = 10ng/mL).
    End point type
    Secondary
    End point timeframe
    24 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    9
    Units: ng/mL
    arithmetic mean (standard deviation)
        Prior to dosing
    20.14 ( 28.61 )
        30 min post dose
    615.53 ( 792.91 )
        1 h post dose
    898.61 ( 779.72 )
        2 h post dose
    404.80 ( 296.00 )
        3 h post dose
    151.30 ( 125.29 )
        4 h post dose
    115.93 ( 151.83 )
    No statistical analyses for this end point

    Secondary: Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Week 36

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    End point title
    		 Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Week 36
    End point description
    Values below the LLOQ have been set to half the LLOQ (LLOQ = 10ng/mL).
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    9
    Units: ng/mL
    arithmetic mean (standard deviation)
        Prior to dosing
    89.78 ( 176.45 )
        30 min post dose
    796.29 ( 839.35 )
        1 h post dose
    713.86 ( 453.54 )
        2 h post dose
    298.29 ( 299.24 )
        3 h post dose
    374.16 ( 459.55 )
        4 h post dose
    348.11 ( 479.79 )
    No statistical analyses for this end point

    Secondary: PK Parameters in a Sub-set of Subjects, Cmax

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    End point title
    		 PK Parameters in a Sub-set of Subjects, Cmax
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    12
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Week 0
    1852.13 ( 80.1 )
        Week 12
    954.29 ( 127.9 )
        Week 24
    524.79 ( 281.6 )
        Week 36
    961.86 ( 73.3 )
    No statistical analyses for this end point

    Secondary: PK Parameters Evaluated in a Sub-set of Subjects, Tmax

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    End point title
    		 PK Parameters Evaluated in a Sub-set of Subjects, Tmax
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    12
    Units: h
    median (full range (min-max))
        Week 0
    1.00 (0.5 to 4.0)
        Week 12
    1.50 (0.5 to 4.0)
        Week 24
    1.00 (0.0 to 1.0)
        Week 36
    1.00 (0.5 to 4.0)
    No statistical analyses for this end point

    Secondary: PK Parameters in a Sub-set of Subjects, AUClast

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    End point title
    		 PK Parameters in a Sub-set of Subjects, AUClast
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    12
    Units: h*ng/mL)
    geometric mean (geometric coefficient of variation)
        Week 0
    3018.15 ( 60.5 )
        Week 12
    1704.92 ( 126.6 )
        Week 24
    995.60 ( 213.2 )
        Week 36
    1622.77 ( 49.8 )
    No statistical analyses for this end point

    Secondary: PK Parameters in a Sub-set of Subjects, Accumulation ratio AUC

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    End point title
    		 PK Parameters in a Sub-set of Subjects, Accumulation ratio AUC
    End point description
    End point type
    Secondary
    End point timeframe
    36 weeks
    End point values
    		 C21 100 mg BID
    Number of subjects analysed
    7
    Units: Ratio
    geometric mean (geometric coefficient of variation)
        Week 12
    0.846 ( 120.5 )
        Week 24
    0.453 ( 328.9 )
        Week 36
    0.738 ( 67.9 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Throughout the trial from signing of informed consent until the end-of-trial visit, up to 44 weeks
    Adverse event reporting additional description
    At each visit the subject was asked about AEs in an objective manner, e.g.: "Have you experienced any problems since the last visit?"
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    C21 100 mg BID
    Reporting group description
    		 -

    Serious adverse events
    		 C21 100 mg BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 52 (9.62%)
         number of deaths (all causes)
    2
         number of deaths resulting from adverse events
    2
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Squamous cell carcinoma of the oral cavity
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac failure
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    Idiopathic pulmonary fibrosis
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonitis
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Infections and infestations
    Kidney infection
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Sepsis
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    COVID-19
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Metabolism and nutrition disorders
    Type 2 diabetes
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 2%
    Non-serious adverse events
    		 C21 100 mg BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    32 / 52 (61.54%)
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    4 / 52 (7.69%)
         occurrences all number
    4
    C-reactive protein increased
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    3
    Blood alkaline phosphatase increased
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    2
    Blood glucose increased
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    2
    Alanine aminotransferase abnormal
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    3
    Aspartate aminotransferase abnormal
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    2
    Cardiac disorders
    Bundle branch block left
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    3
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    8
    Lethargy
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    3
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 52 (7.69%)
         occurrences all number
    4
    Influenza like illness
         subjects affected / exposed
    1 / 52 (1.92%)
         occurrences all number
    2
    Gastrointestinal disorders
    Gastro-oesophageal reflux disease
         subjects affected / exposed
    4 / 52 (7.69%)
         occurrences all number
    4
    Diarrhoea
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    6
    Nausea
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 52 (7.69%)
         occurrences all number
    5
    Dyspnoea
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    5
    Idiopathic pulmonary fibrosis
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    6
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    10 / 52 (19.23%)
         occurrences all number
    10
    Rash
         subjects affected / exposed
    4 / 52 (7.69%)
         occurrences all number
    6
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    3
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    2 / 52 (3.85%)
         occurrences all number
    2
    Infections and infestations
    Lower respiratory tract infection
         subjects affected / exposed
    3 / 52 (5.77%)
         occurrences all number
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Mar 2021
    Protocol version 5.0; Global substantial amendment to protocol version 4.0 included the following changes: 12-week efficacy objective was added. Changes of in-and exclusion criteria: Inclusion criterion 5 modified to include a check at visit 2. Exclusion criterion 1 modified to exclude all types of anti-fibrotic treatment. Exclusion criterion 7 narrowed to only exclude concomitant treatment with strong CYP3A4 inhibitors and inducers, and “or equivalent” added after doses of prednisolone. Exclusion criterion 8 modified to only exclude concomitant treatment with sulphasalazine, rosuvastatin and/or high dose BCRP sensitive substrates.
    27 May 2021
    Protocol version 6.0; Global Substantial amendment to protocol version 5.0 included the following changes: Anticipated enrollment changed from 60 subjects in total to 60 subjects with a completed week 12 visit. Inclusion criterion 8 added requiring that subjects are fully vaccinated against COVID-19 prior to visit 1. Exclusion criterion 5 moderate to severe hepatic impairment added. Exclusion criterion 9 updated to clarify that only subjects with clinically significant cardiac arrhythmias should be excluded. Criterion 9 further updated to exclude subjects with increased AST, AST or bilirubin values. 5.5.2 Withdrawal from Trial Replacement subjects added for subjects withdrawing prior to week 12 until 60 subjects have completed week 12. 6.2 Number of Subjects Anticipated enrollment changed from 60 subjects in total to 60 subjects with a completed week 12 visit. 6.5.1 Screening (Visit 1) Check of COVID-19 vaccination status added to visit 1 procedure. 9.3 Reporting of Adverse Events For AEs concerning hair loss, additional data collection added.
    15 Sep 2021
    Protocol version 7.0; Global Substantial amendment to protocol version 6.0 included the following changes: Inclusion criterion 1 modified from a diagnosis of IPF within 3 years to a diagnosis of IPF within 5 years prior to visit 1. Inclusion criterion 4 modified to allow an FVC of >60 % predicted in UK subjects who have discontinued antifibrotic treatment or refused such treatments. Exclusion criterion 1 updated to allow subjects who have been treated with antifibrotics for ≤6 months. Antifibrotic treatment within 4 weeks prior to visit 1 added to exclusion criterion 7 and disallowed medication Exclusion criterion 18 added to exclude subjects discontinuing or changing antifibrotic treatment due to disease progression.

    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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