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    Clinical Trial Results:
    An open-label, non-randomized study on efficacy, pharmacokinetics, pharmacodynamics, safety and tolerability of LNP023 in two patient populations with C3 glomerulopathy

    Summary
    EudraCT number
    		 2017-000889-29
    Trial protocol
    		 GB   ES   DE   FR   IT  
    Global end of trial date
    23 Apr 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 May 2022
    First version publication date
    08 May 2022
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    CLNP023X2202
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03832114
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharmaceuticals
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111, Novartis.email@novartis.com
    Scientific contact
    Study Director, Novartis Pharmaceuticals, +41 8627788300, Novartis.email@novartis.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
    23 Apr 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Apr 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this trial was to evaluate the efficacy of LNP023 in reducing proteinuria at Week 12 (Cohort A) and to assess histopathological changes in kidney biopsies at Week 12 (Cohort B).
    Protection of trial subjects
    This study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Feb 2019
    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
    France: 6
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    United States: 4
    Worldwide total number of subjects
    27
    EEA total number of subjects
    15
    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)
    25
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 27 patients (16 patients in Cohort A and 11 patients in Cohort B) were enrolled and treated in the treatment period 1. All 27 patients completed the study and there were no patients who discontinued the study.

    Pre-assignment
    Screening details
    		 A total of 27 patients (16 patients in Cohort A and 11 patients in Cohort B) were enrolled and treated in the treatment period 1. All 27 patients completed the study and there were no patients who discontinued the study.

    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
    Yes

    Arm title
    		 Cohort A - no kidney transplant
    Arm description
    		 C3G patients who have not received a kidney transplant and have reduced C3 blood levels.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LNP023
    Investigational medicinal product code
    LNP023
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    200 mg

    Arm title
    		 Cohort B - kidney transplant
    Arm description
    		 C3G patients who have received a kidney transplant and have C3G recurrence.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LNP023
    Investigational medicinal product code
    LNP023
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    200 mg

    Number of subjects in period 1
    		Cohort A - no kidney transplant Cohort B - kidney transplant
    Started
    16
    11
    Completed
    16
    11

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort A - no kidney transplant
    Reporting group description
    		 C3G patients who have not received a kidney transplant and have reduced C3 blood levels.

    Reporting group title
    Cohort B - kidney transplant
    Reporting group description
    		 C3G patients who have received a kidney transplant and have C3G recurrence.

    Reporting group values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant Total
    Number of subjects
    		 16 11 27
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 16 9 25
        From 65-84 years
    		 0 2 2
        85 years and over
    		 0 0 0
    Age Continuous
    Age in Years
    Units: Years
        median (full range (min-max))
    		 22.0 (18 to 59) 31.0 (18 to 70) -
    Sex: Female, Male
    Units: Participants
        Female
    		 6 3 9
        Male
    		 10 8 18
    Race/Ethnicity, Customized
    Units: Subjects
        American Indian or Alaska
    		 0 1 1
        Black or African American
    		 0 1 1
        White
    		 16 9 25
    Subject analysis sets

    Subject analysis set title
    Cohort A
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Summary statistics of PK parameters after multiple dose administration of LNP023 in patients in Cohorts A and B

    Subject analysis set title
    Cohort B
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Summary statistics of PK parameters after multiple dose administration of LNP023 in patients in Cohorts A and B

    Subject analysis sets values
    		 Cohort A Cohort B
    Number of subjects
    16
    11
    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)
    16
    9
        From 65-84 years
    0
    2
        85 years and over
    0
    0
    Age Continuous
    Age in Years
    Units: Years
        median (full range (min-max))
    Sex: Female, Male
    Units: Participants
        Female
        Male
    Race/Ethnicity, Customized
    Units: Subjects
        American Indian or Alaska
        Black or African American
        White

    End points

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    End points reporting groups
    Reporting group title
    Cohort A - no kidney transplant
    Reporting group description
    		 C3G patients who have not received a kidney transplant and have reduced C3 blood levels.

    Reporting group title
    Cohort B - kidney transplant
    Reporting group description
    		 C3G patients who have received a kidney transplant and have C3G recurrence.

    Subject analysis set title
    Cohort A
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Summary statistics of PK parameters after multiple dose administration of LNP023 in patients in Cohorts A and B

    Subject analysis set title
    Cohort B
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Summary statistics of PK parameters after multiple dose administration of LNP023 in patients in Cohorts A and B

    Primary: Cohort A: Change from baseline in Urine Protein to Creatinine concentration Ratio (UPCR)

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    End point title
    		 Cohort A: Change from baseline in Urine Protein to Creatinine concentration Ratio (UPCR) [1]
    End point description
    Change in proteinuria assessed by ratio to baseline of UPCR derived from 24h urine collection
    End point type
    Primary
    End point timeframe
    Week 12
    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: Single arm comparison, therefore no comparison.
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    0 [2]
    Units: g/mol
        geometric mean (confidence interval 80%)
    0.55 (0.46 to 0.65)
    ( to )
    Notes
    [2] - This endpoint is presenting Cohort A only. Cohort B is presented in a separate table.
    No statistical analyses for this end point

    Primary: Cohort B: Change from baseline in C3 Deposit

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    End point title
    		 Cohort B: Change from baseline in C3 Deposit [3]
    End point description
    Histopathological changes in kidney biopsies as assessed by change from baseline in C3 Deposit Score (based on immunofluorescence microscopy)
    End point type
    Primary
    End point timeframe
    Week 12
    Notes
    [3] - 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: Single arm comparison, therefore no comparison.
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    0 [4]
    7
    Units: Percentage
        median (confidence interval 80%)
    ( to )
    -2.50 (-3.75 to -0.75)
    Notes
    [4] - This endpoint is presenting Cohort B only. Cohort A is presented in a separate table.
    No statistical analyses for this end point

    Secondary: Change from baseline in Urine Protein Creatinine Concentration Ratio (UPCR)

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    End point title
    		 Change from baseline in Urine Protein Creatinine Concentration Ratio (UPCR)
    End point description
    Ratio to baseline UPCR derived from 24 hour urine collection
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    7
    Units: 24 hr g/mol
        geometric mean (confidence interval 80%)
    0.55 (0.46 to 0.65)
    0.79 (0.49 to 1.28)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 [5]
    P-value
    		 = 0.0003
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.55
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.46
         upper limit
    		 0.65
    Notes
    [5] - Statistical Analysis is presented below for Cohort A only. Cohort B is presented in a separate table.
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 [6]
    P-value
    		 = 0.4766
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.79
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.49
         upper limit
    		 1.28
    Notes
    [6] - Statistical Analysis is presented for Cohort B below. Cohort A is in a separate table.
    Statistical analysis title
    		 Overall Analysis
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0002
    Method
    		 Mixed Model of Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.59
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.51
         upper limit
    		 0.69

    Secondary: Change from baseline in Urine Protein (UP) Excretion

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    End point title
    		 Change from baseline in Urine Protein (UP) Excretion
    End point description
    Ratio to baseline UP excretion derived from 24 hour urine collection
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    9
    Units: 24 h mg/day
        geometric mean (confidence interval 80%)
    0.57 (0.47 to 0.68)
    1.00 (0.75 to 1.33)
    Statistical analysis title
    		 Overall Analysis
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [7]
    P-value
    		 = 0.0011
    Method
    		 Mixed Model Repeated Measures (MMRM
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.57
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.47
         upper limit
    		 0.68
    Notes
    [7] - Statistical Analysis is presented for Both Arms Overall Values.
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [8]
    P-value
    		 = 0.9998
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.75
         upper limit
    		 1.33
    Notes
    [8] - Statistical Analysis presented for Cohort B below only. Cohort A is presented in a separate table.
    Statistical analysis title
    		 Overall Study
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0016
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.66
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.56
         upper limit
    		 0.77

    Secondary: Change from baseline in Urine Albumin Creatinine concentration Ratio (UACR) Excretion

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    End point title
    		 Change from baseline in Urine Albumin Creatinine concentration Ratio (UACR) Excretion
    End point description
    Ratio to baseline UACR excretion derived from 24 hour urine collection
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    7
    Units: 24 hr g/mol
        geometric mean (confidence interval 80%)
    0.55 (0.47 to 0.64)
    0.61 (0.30 to 1.27)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 [9]
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures (MRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.55
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.47
         upper limit
    		 0.64
    Notes
    [9] - Statistical Analysis presented for Cohort A below only. Cohort B is presented in a separate table.
    Statistical analysis title
    		 Overall Study
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0002
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.6
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.51
         upper limit
    		 0.71
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 [10]
    P-value
    		 = 0.3707
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.61
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.3
         upper limit
    		 1.27
    Notes
    [10] - Statistical Analysis presented for Cohort B only below. Cohort A is presented in a separate table.

    Secondary: Change from baseline change in Urinary Albumin (UA) Excretion

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    End point title
    		 Change from baseline change in Urinary Albumin (UA) Excretion
    End point description
    Ratio to baseline UA excretion derived from 24 hour urine collection
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    9
    Units: 24 h mg/day
        geometric mean (confidence interval 80%)
    0.57 (0.47 to 0.70)
    0.81 (0.67 to 0.98)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [11]
    P-value
    		 = 0.0018
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.57
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.47
         upper limit
    		 0.7
    Notes
    [11] - Statistical Analysis is presented for Cohort A below only. Cohort A is presented in a separate table.
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [12]
    P-value
    		 = 0.1632
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.81
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.67
         upper limit
    		 0.98
    Notes
    [12] - Statistical Analysis is presented below for Cohort B only. Cohort A is presented in a separate table.
    Statistical analysis title
    		 Overall Study
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.004
    Method
    		 Mixed Model Repeated Measure (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.67
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.57
         upper limit
    		 0.79

    Secondary: Change from baseline in estimated glomerular filtration rate (eGFR)

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    End point title
    		 Change from baseline in estimated glomerular filtration rate (eGFR)
    End point description
    Effect of LNP023 on estimated glomerular filtration rate (eGFR)
    End point type
    Secondary
    End point timeframe
    Day 7, Day 28, Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    9
    Units: ml/min
        arithmetic mean (confidence interval 80%)
    2.59 (0.12 to 5.06)
    -0.61 (-3.36 to 2.15)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [13]
    P-value
    		 = 0.1795
    Method
    		 Mixed Model of Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    2.59
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.12
         upper limit
    		 5.06
    Notes
    [13] - Statistical analysis is presented for Cohort A below only. Cohort B is presented in a separate table.
    Statistical analysis title
    		 Overall
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.3754
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.32
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -0.59
         upper limit
    		 3.22
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [14]
    P-value
    		 = 0.7763
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.61
    Confidence interval
         level
    		 0.78%
         sides
    2-sided
         lower limit
    		 -3.36
         upper limit
    		 2.15
    Notes
    [14] - Statistical Analysis presented for Cohort B below only. Cohort A is presented in a separate table .

    Secondary: Change from baseline in serum creatinine

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    End point title
    		 Change from baseline in serum creatinine
    End point description
    The effect of LNP023 on renal function - serum creatinine
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    9
    Units: mmol/L
        arithmetic mean (confidence interval 80%)
    -5.04 (-9.36 to -0.72)
    7.17 (-1.79 to 16.13)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [15]
    P-value
    		 = 0.1364
    Method
    		 Mixed Model Repeated Measuers (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -5.04
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -9.36
         upper limit
    		 -0.72
    Notes
    [15] - Statistical Analysis is presented for Cohort A below only. Cohort B is presented in a separate table.
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 [16]
    P-value
    		 = 0.3038
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    7.17
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -1.79
         upper limit
    		 16.13
    Notes
    [16] - This analysis represents only Cohort B, as Cohort A is in a separate table.
    Statistical analysis title
    		 Overall
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.8352
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.77
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 -5.56
         upper limit
    		 4.01

    Secondary: Change from baseline in creatinine clearance

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    End point title
    		 Change from baseline in creatinine clearance
    End point description
    The effect of LNP023 on renal function - creatinine clearance
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    8
    Units: mL/min
        geometric mean (confidence interval 80%)
    1.07 (0.99 to 1.17)
    1.20 (0.83 to 1.72)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 [17]
    P-value
    		 = 0.2752
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.07
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.99
         upper limit
    		 1.17
    Notes
    [17] - The analysis only represents Cohort A, as Cohort B is presented in a separate table .
    Statistical analysis title
    		 Overall
    Statistical analysis description
    Overall- Day 84
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.1963
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.1
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 1
         upper limit
    		 1.2
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 84
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 [18]
    P-value
    		 = 0.476
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.2
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.83
         upper limit
    		 1.72
    Notes
    [18] - Statistical analysis presented for Cohort B only. Cohort A is presented in a separate table.

    Secondary: Change from baseline and evolution of hematuria

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    End point title
    		 Change from baseline and evolution of hematuria
    End point description
    The effect of LNP023 on renal function - hematuria
    End point type
    Secondary
    End point timeframe
    Week 12: Day 84
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    9
    Units: rbc/hpf
        Week 12: Day 84: <9 rbc/hpf n (%)
    10
    3
        Week 12: Day 84: >= 9 to =<50 rbc/hpf n (%)
    0
    0
        Week 12: Day 84: >50 rbc/hpf n (%)
    0
    0
    No statistical analyses for this end point

    Secondary: Change from baseline in Urine Protein to Creatinine Concentration Ratio (UPCR) First Morning Void

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    End point title
    		 Change from baseline in Urine Protein to Creatinine Concentration Ratio (UPCR) First Morning Void
    End point description
    Ratio to baseline of UPCR reduction derived from total cumulative urinary excretion first morning void
    End point type
    Secondary
    End point timeframe
    Week 9: Day 64
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    15
    7
    Units: g/mol
        geometric mean (confidence interval 80%)
    0.56 (0.48 to 0.65)
    0.99 (0.76 to 1.28)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 64
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 [19]
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.56
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.48
         upper limit
    		 0.65
    Notes
    [19] - Statistical Analysis is presented for Cohort A below. Cohort B is in a separate table .
    Statistical analysis title
    		 Overall Analysis
    Statistical analysis description
    Overall- Day 64
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.64
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.56
         upper limit
    		 0.72
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 64
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 [20]
    P-value
    		 = 0.9544
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.99
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.76
         upper limit
    		 1.28
    Notes
    [20] - Statistical Analysis is presented for Cohort B below. Cohort A is presented in a separate table .

    Secondary: Change from baseline in Urine Albumin to Creatinine Concentration Ratio (UACR) First Morning Void

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    End point title
    		 Change from baseline in Urine Albumin to Creatinine Concentration Ratio (UACR) First Morning Void
    End point description
    UACR reduction derived from total cumulative urinary excretion first morning void
    End point type
    Secondary
    End point timeframe
    Week 9: Day 64
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    15
    7
    Units: g/mol
        geometric mean (confidence interval 80%)
    0.59 (0.50 to 0.69)
    0.87 (0.60 to 1.26)
    Statistical analysis title
    		 Cohort A
    Statistical analysis description
    Day 64
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 [21]
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.59
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.5
         upper limit
    		 0.69
    Notes
    [21] - Statistical Analysis is presented below for Cohort A only. Cohort B is presented in a separate table.
    Statistical analysis title
    		 Cohort B
    Statistical analysis description
    Day 64
    Comparison groups
    Cohort B - kidney transplant v Cohort A - no kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    		 [22]
    P-value
    		 = 0.6209
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Median difference (final values)
    Point estimate
    0.87
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.6
         upper limit
    		 1.26
    Notes
    [22] - Statistical Analysis is presented for Cohort B only below. Cohort A is presented in a separate table.
    Statistical analysis title
    		 Overall Analysis
    Statistical analysis description
    Overall- Day 64
    Comparison groups
    Cohort A - no kidney transplant v Cohort B - kidney transplant
    Number of subjects included in analysis
    22
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0002
    Method
    		 Mixed Model Repeated Measures (MMRM)
    Parameter type
    		 Mean difference (final values)
    Point estimate
    0.63
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.54
         upper limit
    		 0.73

    Secondary: Pharmacokinetics of LNP023 Area under the Plasma-concentration-time curve AUClast (AUC)

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    End point title
    		 Pharmacokinetics of LNP023 Area under the Plasma-concentration-time curve AUClast (AUC)
    End point description
    Plasma: Non-compartmental parameter analysis related to total drug for the area under the plasma-concentration-time curve calculated from time. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4
    End point values
    		 Cohort A Cohort B
    Number of subjects analysed
    16
    11
    Units: hr*ng/mL
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics of LNP023 Area under the Plasma-concentration-time curve AUCtau (AUC)

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    End point title
    		 Pharmacokinetics of LNP023 Area under the Plasma-concentration-time curve AUCtau (AUC)
    End point description
    Plasma: Non-compartmental parameter analysis related to total drug for the area under the plasma-concentration-time curve calculated to the end of the dosing interval (ngxh/mL). Data will be provided at at later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4
    End point values
    		 Cohort A Cohort B
    Number of subjects analysed
    16
    11
    Units: hr*ng/mL
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Observed maximum concentration after drug administration (Cmax)

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    End point title
    		 Observed maximum concentration after drug administration (Cmax)
    End point description
    Plasma: Non-compartmental parameter analysis related to total drug, including but not limited to Cmax after the first dose. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4
    End point values
    		 Cohort A Cohort B
    Number of subjects analysed
    16
    11
    Units: ng/ML
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Observed mimum concentration after drug administration (Ctrough)

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    End point title
    		 Observed mimum concentration after drug administration (Ctrough)
    End point description
    Plasma: Non-compartmental parameter analysis related to total drug, including but not limited to Ctrough (Cmin) after the first dose. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4
    End point values
    		 Cohort A Cohort B
    Number of subjects analysed
    16
    11
    Units: ng/mL
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Time to reach the maximum plasma concentration (Tmax)

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    End point title
    		 Time to reach the maximum plasma concentration (Tmax)
    End point description
    Plasma: Non-compartmental parameter analysis related to total drug, including but not limited to Tmax after the first dose. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    11
    Units: hour (hr)
        median (full range (min-max))
    0 (0 to 0)
    0 (0 to 0)
    No statistical analyses for this end point

    Secondary: Summary of Change from Baseline Complement C3 biomarker in serum

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    End point title
    		 Summary of Change from Baseline Complement C3 biomarker in serum
    End point description
    To assess the effect of LNP023 on alternative complement pathway hyperactivity. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 3, 4, 12
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    10
    Units: g/L
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Ratio to baseline summary of Plasma Bb in blood and urine

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    End point title
    		 Ratio to baseline summary of Plasma Bb in blood and urine
    End point description
    To assess the relationship between LNP023 dose and pharmacodynamic biomarker levels of blood Bb in blood and urine. Data will be provided at a later date.
    End point type
    Secondary
    End point timeframe
    Week 1, 2, 3, 4 and 12
    End point values
    		 Cohort A - no kidney transplant Cohort B - kidney transplant
    Number of subjects analysed
    16
    10
    Units: ng/mL
        arithmetic mean (standard deviation)
    0 ( 0 )
    0 ( 0 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment-emergent adverse events
    Adverse event reporting additional description
    Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Cohort A - Run-in Phase
    Reporting group description
    		 Cohort A - Run-in Phase

    Reporting group title
    Cohort A - Dose Escalation Phase
    Reporting group description
    		 Cohort A - Dose Escalation Phase

    Reporting group title
    Cohort B - Dose Escalation Phase
    Reporting group description
    		 Cohort B - Dose Escalation Phase

    Reporting group title
    Cohort B - Run-in Phase
    Reporting group description
    		 Cohort B - Run-in Phase

    Reporting group title
    Cohort B - LNP023 200mg b.i.d.
    Reporting group description
    		 Cohort B - LNP023 200mg b.i.d.

    Reporting group title
    Cohort A - LNP023 200mg b.i.d.
    Reporting group description
    		 Cohort A - LNP023 200mg b.i.d.

    Serious adverse events
    		 Cohort A - Run-in Phase Cohort A - Dose Escalation Phase Cohort B - Dose Escalation Phase Cohort B - Run-in Phase Cohort B - LNP023 200mg b.i.d. Cohort A - LNP023 200mg b.i.d.
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    2 / 11 (18.18%)
    0 / 16 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Overdose
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort A - Run-in Phase Cohort A - Dose Escalation Phase Cohort B - Dose Escalation Phase Cohort B - Run-in Phase Cohort B - LNP023 200mg b.i.d. Cohort A - LNP023 200mg b.i.d.
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    6 / 16 (37.50%)
    5 / 11 (45.45%)
    0 / 11 (0.00%)
    6 / 11 (54.55%)
    8 / 16 (50.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Spider vein
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Oedema peripheral
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Psychiatric disorders
    Adjustment disorder with depressed mood
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Intentional self-injury
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    0
    0
    1
    1
    Blood luteinising hormone increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Body temperature increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Lipase increased
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Nervous system disorders
    Anosmia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Dysgeusia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Headache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    2 / 11 (18.18%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    Migraine
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Syncope
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Normochromic normocytic anaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Eye disorders
    Conjunctival hyperaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    Dyspepsia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Skin discolouration
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Hordeolum
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Influenza
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Dyslipidaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gout
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Hypercholesterolaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 16 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Hyperkalaemia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    2 / 11 (18.18%)
    0 / 16 (0.00%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    Iron deficiency
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
    1 / 16 (6.25%)
         occurrences all number
    0
    0
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Nov 2018
    Non-Substantial Amendment: text added to indicate patients must receive vaccinations against N. meningitidis, S. pneumoniae and H. influenzae prior to first dosing.
    22 Feb 2019
    Germany: The purpose of this local protocol amendment was to modify the study stopping rules for drug related events and to clarify if the study was halted the restart would be documented by a substantial amendment as per Health Authority questions.
    20 Aug 2019
    The purpose of this amendment was to include a second treatment period (Treatment period 2) to enable the prolongation of treatment by 12 weeks for any patient.
    07 Feb 2020
    The purpose of this amendment was mainly to correct the estimation of the blood sample volume to be collected, confirming that there is no increased risk for the patient.

    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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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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