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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo Controlled Study to Evaluate Efficacy and Safety of Nefecon in Patients with Primary IgA Nephropathy at Risk of Progressing to End-Stage Renal Disease (NefIgArd).

    Summary
    EudraCT number
    		 2017-004902-16
    Trial protocol
    		 CZ   SE   BE   FI   ES   GB   PL   IT  
    Global end of trial date
    19 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Jul 2024
    First version publication date
    03 Jul 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    Nef-301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03643965
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Calliditas Therapeutics AB
    Sponsor organisation address
    Kungsbron 1, Stockholm, Sweden, 111 22
    Public contact
    Clinical Operations, Calliditas Therapeutics AB, kristin.onnestam@calliditas.com
    Scientific contact
    Clinical Operations, Calliditas Therapeutics AB, kristin.onnestam@calliditas.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
    08 Nov 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Part A: The primary objective of Part A is to assess the effect of Nefecon 16 mg treatment on urine protein to creatinine ratio (UPCR) over 9 months compared to placebo. Part B: The primary objective of Part B is to assess the effect of the Nefecon 16 mg treatment given in Part A on clinical consequences of any proteinuria reduction as measured by estimated glomerular filtration rate (eGFR) recorded over 2 years compared to placebo.
    Protection of trial subjects
    A Data Safety Monitoring Board (DSMB) was established to review and discuss the available study data as patients were randomized and followed throughout the study. The DSMB also acted as an expert, independent advisory to study conduct. The DSMB met periodically throughout the course of the study to review unblinded safety data.
    Background therapy
    		 Optimized renin angiotensin system (RAS) inhibitor therapy
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 May 2018
    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
    Poland: 7
    Country: Number of subjects enrolled
    Spain: 8
    Country: Number of subjects enrolled
    Sweden: 10
    Country: Number of subjects enrolled
    United Kingdom: 19
    Country: Number of subjects enrolled
    Belgium: 14
    Country: Number of subjects enrolled
    Czechia: 31
    Country: Number of subjects enrolled
    Finland: 7
    Country: Number of subjects enrolled
    France: 10
    Country: Number of subjects enrolled
    Germany: 33
    Country: Number of subjects enrolled
    Greece: 24
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    Argentina: 18
    Country: Number of subjects enrolled
    Australia: 21
    Country: Number of subjects enrolled
    Belarus: 7
    Country: Number of subjects enrolled
    Canada: 24
    Country: Number of subjects enrolled
    China: 33
    Country: Number of subjects enrolled
    Korea, Democratic People's Republic of: 20
    Country: Number of subjects enrolled
    Taiwan: 2
    Country: Number of subjects enrolled
    Türkiye: 21
    Country: Number of subjects enrolled
    United States: 49
    Worldwide total number of subjects
    364
    EEA total number of subjects
    150
    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)
    353
    From 65 to 84 years
    11
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Global multi-center study. Randomized.

    Pre-assignment
    Screening details
    		 Screening period up to 35 days prior to enrollment

    Period 1
    Period 1 title
    Part A
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Nefecon 16 mg
    Arm description
    		 Nefecon 16 mg once daily for 9 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Nefecon
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    16 mg (4 capsules) once daily by mouth

    Arm title
    		 Placebo
    Arm description
    		 Placebo once daily for 9 months
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo (4 capsules) once daily for 9 months.

    Number of subjects in period 1
    		Nefecon 16 mg Placebo
    Started
    182
    182
    Completed
    175
    174
    Not completed
    7
    8
         All
    7
    8
    Period 2
    Period 2 title
    Part B
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Part B was an observation period without treatment. Blinding to treatment in Part A applied.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Nefecon 16 mg
    Arm description
    		 No treatment in Part B but observation following Part A treatment.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Nefecon
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    16 mg (4 capsules) once daily by mouth

    Arm title
    		 Placebo
    Arm description
    		 No treatment in Part B but observation following Part A treatment.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo (4 capsules) once daily for 9 months.

    Number of subjects in period 2
    		Nefecon 16 mg Placebo
    Started
    175
    174
    Completed
    161
    165
    Not completed
    14
    9
         All
    14
    9

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Nefecon 16 mg
    Reporting group description
    		 Nefecon 16 mg once daily for 9 months.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo once daily for 9 months

    Reporting group values
    		 Nefecon 16 mg Placebo Total
    Number of subjects
    		 182 182 364
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 174 179 353
        From 65-84 years
    		 8 3 11
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 43.8 ( 10.78 ) 41.6 ( 10.65 ) -
    Gender categorical
    Units: Subjects
        Female
    		 65 59 124
        Male
    		 117 123 240
    Baseline UPCR
    Units: g/gram
        geometric mean (inter-quartile range (Q1-Q3))
    		 1.300 (0.902 to 1.764) 1.264 (0.877 to 1.752) -
    Baseline eGFR
    Units: ml/min/1.73m2
        geometric mean (inter-quartile range (Q1-Q3))
    		 56.006 (45.497 to 70.972) 55.565 (45.957 to 67.735) -

    End points

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    End points reporting groups
    Reporting group title
    Nefecon 16 mg
    Reporting group description
    		 Nefecon 16 mg once daily for 9 months.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo once daily for 9 months
    Reporting group title
    Nefecon 16 mg
    Reporting group description
    		 No treatment in Part B but observation following Part A treatment.

    Reporting group title
    Placebo
    Reporting group description
    		 No treatment in Part B but observation following Part A treatment.

    Primary: Part A: Ratio of UPCR at 9 Months Compared to Baseline

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    End point title
    		 Part A: Ratio of UPCR at 9 Months Compared to Baseline
    End point description
    End point type
    Primary
    End point timeframe
    9 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    97
    102
    Units: g/gram
        least squares mean (confidence interval 96%)
    0.69 (0.61 to 0.79)
    0.95 (0.83 to 1.08)
    Statistical analysis title
    		 Part A: Ratio of UPCR at 9 Months Compared to Base
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    199
    Analysis specification
    Pre-specified
    Analysis type
    		 other [1]
    P-value
    		 = 0.0003
    Method
    		 MMRM model
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    0.73
    Confidence interval
         level
    		 96%
         sides
    2-sided
         lower limit
    		 0.61
         upper limit
    		 0.88
    Notes
    [1] - Ratio of UPCR at 9 months compared to baseline for Nefecon compared to Placebo

    Primary: Part B: Time-weighted Average of eGFR

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    End point title
    		 Part B: Time-weighted Average of eGFR
    End point description
    End point type
    Primary
    End point timeframe
    Up to 2 years
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    182
    182
    Units: mL/min/1.73m2
        least squares mean (confidence interval 95%)
    0.96 (0.93 to 0.98)
    0.87 (0.84 to 0.89)
    Statistical analysis title
    		 Time-weighted average of eGFR, Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    364
    Analysis specification
    Pre-specified
    Analysis type
    		 other [2]
    P-value
    		 < 0.0001
    Method
    		 robust regression
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    1.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.06
         upper limit
    		 1.15
    Notes
    [2] - Time-weighted average of eGFR

    Secondary: Part A: Ratio of eGFR at 9 Months

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    End point title
    		 Part A: Ratio of eGFR at 9 Months
    End point description
    End point type
    Secondary
    End point timeframe
    9 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    97
    102
    Units: mL/min/1.73m2
        least squares mean (confidence interval 95%)
    1.00 (0.96 to 1.03)
    0.93 (0.90 to 0.96)
    Statistical analysis title
    		 Ratio of eGFR at 9 months Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    199
    Analysis specification
    Pre-specified
    Analysis type
    		 other [3]
    P-value
    		 = 0.0014
    Method
    		 robust regression
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    1.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.03
         upper limit
    		 1.13
    Notes
    [3] - Ratio of eGFR at 9 months comparison of Nefecon to Placebo

    Secondary: Part A: Ratio of eGFR at 12 Months

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    End point title
    		 Part A: Ratio of eGFR at 12 Months
    End point description
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    97
    102
    Units: mL/min/1.73m2
        least squares mean (confidence interval 95%)
    0.97 (0.93 to 1.01)
    0.91 (0.88 to 0.95)
    Statistical analysis title
    		 Ratio of eGFR at 12 months Nefecon vs Placebo
    Comparison groups
    Placebo v Nefecon 16 mg
    Number of subjects included in analysis
    199
    Analysis specification
    Pre-specified
    Analysis type
    		 other [4]
    P-value
    		 = 0.0106
    Method
    		 robust regression
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    1.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.01
         upper limit
    		 1.13
    Notes
    [4] - Ratio of eGFR at 12 months comparison of Nefecon to Placebo

    Secondary: Part A: Ratio of UACR at 9 Months

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    End point title
    		 Part A: Ratio of UACR at 9 Months
    End point description
    End point type
    Secondary
    End point timeframe
    9 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    97
    102
    Units: g/gram
        least squares mean (confidence interval 95%)
    0.64 (0.55 to 0.75)
    0.93 (0.80 to 1.09)
    Statistical analysis title
    		 Ratio of UACR at 9 months Nefecon vs Placebo
    Comparison groups
    Placebo v Nefecon 16 mg
    Number of subjects included in analysis
    199
    Analysis specification
    Pre-specified
    Analysis type
    		 other [5]
    P-value
    		 = 0.0005
    Method
    		 MMRM model
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    0.69
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.55
         upper limit
    		 0.86
    Notes
    [5] - Ratio of UACR at 9 months comparison of Nefecon to Placebo

    Secondary: Part B: Time to 30% Reduction in eGFR

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    End point title
    		 Part B: Time to 30% Reduction in eGFR
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 2 years
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    182
    182
    Units: Participants
    21
    39
    Statistical analysis title
    		 Time to 30% reduction in eGFR Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    364
    Analysis specification
    Pre-specified
    Analysis type
    		 other [6]
    P-value
    		 = 0.0028
    Method
    		 Cox proportional hazards model
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.45
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.26
         upper limit
    		 0.75
    Notes
    [6] - Time to 30% reduction in eGFR comparison Nefecon to Placebo. Cox proportional hazards model with individual patient censoring weighting.

    Secondary: Part B: Time to Receiving Rescue Medication.

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    End point title
    		 Part B: Time to Receiving Rescue Medication.
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 2 years
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    182
    182
    Units: Participants
    15
    20
    Statistical analysis title
    		 Time to receiving rescue medication comparison
    Statistical analysis description
    Time to receiving rescue medication comparison Nefecon to Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    364
    Analysis specification
    Pre-specified
    Analysis type
    		 other [7]
    P-value
    		 = 0.2647
    Method
    		 Cox proportional hazards model
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.68
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.34
         upper limit
    		 1.33
    Notes
    [7] - Time to receiving rescue medication comparison Nefecon to Placebo

    Secondary: Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months

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    End point title
    		 Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
    End point description
    End point type
    Secondary
    End point timeframe
    12 to 24 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    172
    173
    Units: g/gram
        least squares mean (confidence interval 95%)
    0.60 (0.54 to 0.66)
    1.01 (0.91 to 1.12)
    Statistical analysis title
    		 Ratio of UPCR compared to baseline
    Statistical analysis description
    Ratio of UPCR compared to baseline averaged over time points between 12 and 24 months, comparison Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    345
    Analysis specification
    Pre-specified
    Analysis type
    		 other [8]
    P-value
    		 < 0.0001
    Method
    		 MMRM model
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    0.59
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.51
         upper limit
    		 0.68
    Notes
    [8] - Ratio of UPCR compared to baseline averaged over time points between 12 and 24 months, comparison Nefecon vs Placebo

    Secondary: Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months

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    End point title
    		 Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
    End point description
    End point type
    Secondary
    End point timeframe
    12 to 24 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    172
    173
    Units: g/gram
        least squares mean (confidence interval 95%)
    0.52 (0.46 to 0.58)
    0.96 (0.86 to 1.08)
    Statistical analysis title
    		 Ratio of UACR compared to baseline
    Statistical analysis description
    Ratio of UACR compared to baseline averaged over time points between 12 and 24 months, Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    345
    Analysis specification
    Pre-specified
    Analysis type
    		 other [9]
    P-value
    		 < 0.0001
    Method
    		 MMRM model
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    0.54
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.45
         upper limit
    		 0.63
    Notes
    [9] - Ratio of UACR compared to baseline averaged over time points between 12 and 24 months, Nefecon vs Placebo

    Secondary: Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months

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    End point title
    		 Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
    End point description
    End point type
    Secondary
    End point timeframe
    12 to 24 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    163
    166
    Units: mL/min/1.73m2
        least squares mean (confidence interval 95%)
    0.93 (0.90 to 0.96)
    0.84 (0.81 to 0.86)
    Statistical analysis title
    		 Ratio of eGFR compared to baseline
    Statistical analysis description
    Ratio of eGFR compared to baseline averaged over time points between 12 and 24 months, comparison Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    329
    Analysis specification
    Pre-specified
    Analysis type
    		 other [10]
    P-value
    		 < 0.0001
    Method
    		 robust regression
    Parameter type
    		 Ratio of geometric LS means
    Point estimate
    1.11
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.06
         upper limit
    		 1.16
    Notes
    [10] - Ratio of eGFR compared to baseline averaged over time points between 12 and 24 months, comparison Nefecon vs Placebo

    Secondary: Part B: Proportion of Patients Without Microhematuria

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    End point title
    		 Part B: Proportion of Patients Without Microhematuria
    End point description
    End point type
    Secondary
    End point timeframe
    12 to 24 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    182
    182
    Units: Participants
    94
    59
    Statistical analysis title
    		 Proportion of patients without microhematuria
    Statistical analysis description
    Proportion of patients without microhematuria, comparison Nefecon vs Placebo
    Comparison groups
    Nefecon 16 mg v Placebo
    Number of subjects included in analysis
    364
    Analysis specification
    Pre-specified
    Analysis type
    		 other [11]
    P-value
    		 = 0.0001
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.6
         upper limit
    		 4.1
    Notes
    [11] - Proportion of patients without microhematuria, comparison Nefecon vs Placebo

    Secondary: Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 months

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    End point title
    		 Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 months
    End point description
    End point type
    Secondary
    End point timeframe
    9 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    170
    170
    Units: score
    arithmetic mean (standard deviation)
        Bodily Pain
    54.353 ( 9.0306 )
    54.659 ( 9.2387 )
        General Health
    47.058 ( 9.6373 )
    48.014 ( 10.0120 )
        Mental Component Summary
    49.564 ( 9.2834 )
    49.760 ( 8.8974 )
        Mental Health
    50.226 ( 8.9539 )
    50.099 ( 8.8483 )
        Physical Component Summary
    52.257 ( 7.2188 )
    53.732 ( 6.5876 )
        Physical Functioning
    52.722 ( 6.7370 )
    54.276 ( 5.2243 )
        Role Emotional
    49.574 ( 9.4322 )
    50.393 ( 8.3001 )
        Role Physical
    51.028 ( 8.0207 )
    52.693 ( 6.6600 )
        Social Function
    50.941 ( 8.3606 )
    51.707 ( 7.9259 )
        Vitality
    51.826 ( 10.0909 )
    53.490 ( 9.3987 )
    No statistical analyses for this end point

    Secondary: Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 months

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    End point title
    		 Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 months
    End point description
    End point type
    Secondary
    End point timeframe
    24 months
    End point values
    		 Nefecon 16 mg Placebo
    Number of subjects analysed
    159
    164
    Units: score
    arithmetic mean (standard deviation)
        Bodily Pain
    53.385 ( 9.6352 )
    53.208 ( 9.9811 )
        General Health
    47.038 ( 9.3450 )
    47.214 ( 9.9564 )
        Mental Component Summary
    51.246 ( 8.1362 )
    49.743 ( 10.2184 )
        Mental Health
    51.691 ( 8.2104 )
    50.294 ( 10.0007 )
        Physical Component Summary
    51.443 ( 7.5200 )
    51.993 ( 7.1783 )
        Physical Functioning
    52.665 ( 7.6364 )
    53.059 ( 6.9006 )
        Role Emotional
    50.322 ( 8.4690 )
    49.333 ( 10.0789 )
        Role Physical
    50.948 ( 7.8921 )
    50.722 ( 8.8255 )
        Social Functioning
    51.949 ( 7.9677 )
    51.471 ( 8.9304 )
        Vitality
    53.291 ( 8.8558 )
    52.364 ( 10.0023 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs were recorded from time of first dose of study treatment until 2 yrs after first dose (end of study). SAEs from ICF. Treatment Emergent AEs were those that occurred after the first dose of study drug until 14 days after the last dose.
    Adverse event reporting additional description
    Treatment emergent AEs and SAEs are reported below.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Nefecon 16 mg
    Reporting group description
    		 Nefecon 16 mg once daily for 9 months.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo once daily for 9 months

    Serious adverse events
    		 Nefecon 16 mg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    18 / 182 (9.89%)
    11 / 182 (6.04%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Meningioma
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 182 (1.10%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypertensive urgency
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Post thrombotic syndrome
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Abortion spontaneous
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Face oedema
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Generalised oedema
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Oedema peripheral
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    2 / 182 (1.10%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Suicidal ideation
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac failure congestive
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Transient ischaemic attack
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Bone marrow oedema
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastritis haemorrhagic
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis acute
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 182 (0.55%)
    2 / 182 (1.10%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrotic syndrome
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal impairment
         subjects affected / exposed
    2 / 182 (1.10%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Rhabdomyolysis
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Campylobacter colitis
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Coronavirus infection
    Additional description: Treatment emergent
         subjects affected / exposed
    2 / 182 (1.10%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Diverticulitis
         subjects affected / exposed
    0 / 182 (0.00%)
    1 / 182 (0.55%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Erysipelas
         subjects affected / exposed
    1 / 182 (0.55%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 182 (1.10%)
    0 / 182 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Nefecon 16 mg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    108 / 182 (59.34%)
    69 / 182 (37.91%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    21 / 182 (11.54%)
    6 / 182 (3.30%)
         occurrences all number
    24
    6
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    28 / 182 (15.38%)
    7 / 182 (3.85%)
         occurrences all number
    33
    8
    Fatigue
         subjects affected / exposed
    10 / 182 (5.49%)
    7 / 182 (3.85%)
         occurrences all number
    10
    8
    Headache
         subjects affected / exposed
    19 / 182 (10.44%)
    14 / 182 (7.69%)
         occurrences all number
    26
    16
    Weight increased
         subjects affected / exposed
    10 / 182 (5.49%)
    5 / 182 (2.75%)
         occurrences all number
    10
    5
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    9 / 182 (4.95%)
    10 / 182 (5.49%)
         occurrences all number
    9
    11
    Dyspepsia
         subjects affected / exposed
    13 / 182 (7.14%)
    4 / 182 (2.20%)
         occurrences all number
    18
    4
    Nausea
         subjects affected / exposed
    8 / 182 (4.40%)
    12 / 182 (6.59%)
         occurrences all number
    8
    12
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    20 / 182 (10.99%)
    2 / 182 (1.10%)
         occurrences all number
    21
    2
    Rash
         subjects affected / exposed
    10 / 182 (5.49%)
    7 / 182 (3.85%)
         occurrences all number
    13
    12
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    10 / 182 (5.49%)
    7 / 182 (3.85%)
         occurrences all number
    10
    7
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    12 / 182 (6.59%)
    4 / 182 (2.20%)
         occurrences all number
    12
    5
    Muscle spasms
         subjects affected / exposed
    22 / 182 (12.09%)
    7 / 182 (3.85%)
         occurrences all number
    27
    8
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    17 / 182 (9.34%)
    19 / 182 (10.44%)
         occurrences all number
    18
    25
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 182 (5.49%)
    10 / 182 (5.49%)
         occurrences all number
    12
    16

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Nov 2018
    Updated exclusion criteria​ Biopsy limit changed from 5 years to 10 years​ GCS treatment before randomization changed from 2 years to 1 year​ Schedule of procedures related to exploratory sampling changed​ SAE reporting period starts from signature of ICF​ Quality control requirements clarified
    02 Jan 2019
    2 consecutive measurements of proteinuria required ​ The lower limit of the eGFR value was reduced from 45 to 35 mL/min per 1.73 m2​ Schedule of event at study visits was updated​ Clarified that AESIs would be followed until 2 years after the first dose of study drug
    19 Dec 2019
    Updated exclusion criteria​ Primary endpoint Part B eGFR was time-weighted average of AUC over 24 months​ Secondary objectives to assess Nefecon treatment effects with Part A​ China included​ 360 patients to be randomized​ Part B duration changed to 12 months​ Updated exclusion criteria​ AESI’s specified
    28 Apr 2020
    Covid-19 - study treatment up to 10 m possible​ Covid-19 - study procedures – last visit ((V11 Part A) to site changed to by phone
    14 Jan 2021
    Consistency with SAP v 4.0​

    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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